{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,12]],"date-time":"2026-09-12T18:11:16Z","timestamp":1789236676042,"version":"build-2803163510"},"reference-count":22,"publisher":"American Medical Association (AMA)","issue":"13","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA"],"published-print":{"date-parts":[[2025,4,1]]},"abstract":"\n Importance<\/jats:title>\n \n Bioenergetic failure has been proposed as a driver of amyotrophic lateral sclerosis (ALS). CNM-Au8 is a suspension of gold nanocrystals that catalyzes the conversion of nicotinamide adenine dinucleotide hydride into NAD\n +<\/jats:sup>\n , resulting in an increase of cellular adenosine triphosphate production.\n <\/jats:p>\n <\/jats:sec>\n \n Objective<\/jats:title>\n To determine the effects of CNM-Au8 on ALS disease progression.<\/jats:p>\n <\/jats:sec>\n \n Design, Setting, and Participants<\/jats:title>\n CNM-Au8 was tested as a regimen of the HEALEY ALS Platform Trial, a phase 2\/3, multicenter, randomized, double-blind platform trial. The study was conducted at 54 sites in the US from July 2020 to March 2022 (final follow-up, March 17, 2022). A total of 161 participants with ALS were randomized to receive CNM-Au8 (n\u2009=\u2009120) or regimen-specific placebo (n\u2009=\u200941). Data from 123 concurrently randomized placebo participants in other regimens were combined for analyses.<\/jats:p>\n <\/jats:sec>\n \n Interventions<\/jats:title>\n Eligible participants were randomized in a 3:3:2 ratio to receive CNM-Au8 60 mg daily (n\u2009=\u200961), CNM-Au8 30 mg daily (n\u2009=\u200959), or matching placebo (n\u2009=\u200941) for 24 weeks.<\/jats:p>\n <\/jats:sec>\n \n Main Outcomes and Measures<\/jats:title>\n The primary efficacy outcome was change from baseline through week 24 in ALS disease severity measured by a bayesian shared parameter model of function (based on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale) and survival, which provided an estimate of the rate of disease progression measured by the disease rate ratio (DRR), with a DRR of less than 1 indicating treatment benefit. Secondary end points included a Combined Assessment of Function and Survival using a joint-rank test, rate of decline in slow vital capacity (percent predicted), and survival free of permanent assisted ventilation.<\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n Among 161 participants who were randomized within the CNM-Au8 regimen (mean age, 58.4 years; 61 [37.9%] female), 145 (90%) completed the trial. In the primary analysis comparing the combined CNM-Au8 dosage groups vs the combined placebo groups, the primary end point (DRR, 0.97 [95% credible interval, 0.783-1.175]; posterior probability of DRR &amp;lt;1, 0.65) and the 3 secondary end points suggested no benefit or harm of CNM-Au8. In the active (n\u2009=\u2009120) vs placebo (n\u2009=\u2009163) groups, the most common adverse events were diarrhea (23 [19%] vs 12 [7%]), nausea (17 [14.2%] vs 14 [8.6%]), fatigue (12 [10.8%] vs 30 [18.4%]), and muscular weakness (24 [20%] vs 45 [27.6%]).<\/jats:p>\n <\/jats:sec>\n \n Conclusions and Relevance<\/jats:title>\n No benefit of CNM-Au8 on ALS disease progression was observed at 24 weeks.<\/jats:p>\n <\/jats:sec>\n \n Trial Registration<\/jats:title>\n \n ClinicalTrials.gov Identifiers:\n NCT04297683<\/jats:ext-link>\n ,\n NCT04414345<\/jats:ext-link>\n <\/jats:p>\n <\/jats:sec>","DOI":"10.1001\/jama.2024.27643","type":"journal-article","created":{"date-parts":[[2025,2,17]],"date-time":"2025-02-17T11:32:42Z","timestamp":1739791962000},"page":"1138","source":"Crossref","is-referenced-by-count":17,"title":["CNM-Au8 in Amyotrophic Lateral Sclerosis"],"prefix":"10.1001","volume":"333","author":[{"name":"Writing Committee for the HEALEY ALS Platform Trial","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Douglas","family":"Hayden","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Po-Ying","family":"Lai","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachel A.","family":"Donahue","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hao-Wun","family":"Chen","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jianing","family":"Wang","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nithya","family":"Mathai","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabriela","family":"Lopes","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra","family":"McCaffrey","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Scalia","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Luppino","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Clotilde","family":"Lagier-Tourenne","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ghazaleh","family":"Sadri-Vakili","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen","family":"Kolb","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Heintzman","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Sufit","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"April","family":"Szymanski","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liberty","family":"Jenkins","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alan","family":"Martin","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ericka","family":"(Simpson) Greene","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jason R.","family":"Thonhoff","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bing","family":"Liao","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charles","family":"Whitaker","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lora L.","family":"Clawson","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alpa","family":"Uchil","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristen M.","family":"Riley","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"JinAe","family":"Arneklev","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"Grogan","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaowei","family":"Su","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mansoureh","family":"Mamarabadi","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amber","family":"Malcolm","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tracy","family":"Bazan","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nassim","family":"Rad","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leo H.","family":"Wang","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eva L.","family":"Feldman","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ezequiel","family":"Piccione","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pariwat","family":"Thaisetthawatkul","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Constantine","family":"Farmakidis","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Duaa","family":"Jabari","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Statland","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mamatha","family":"Pasnoor","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mazen","family":"Dimachkie","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"Jr","given":"Robert H.","family":"Brown","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mehdi","family":"Ghasemi","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hajar","family":"Houmani","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Douthwright","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kate","family":"Daniello","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Niraja","family":"Suresh","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerrica","family":"Farias","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"I-Hweii A.","family":"Chen","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Piera","family":"Pasinelli","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kara","family":"Steijlen","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ratna","family":"Bhavaraju-Sanka","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bill","family":"Jacobsen","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jourdan","family":"Milliard","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Bowser","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anahita","family":"Deboo","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael S.","family":"Cartwright","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher","family":"Nance","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ludwig","family":"Gutmann","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julia","family":"Yasek","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Harms","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Burford","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frank","family":"Diaz","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Shrilla","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Goran","family":"Rakocevic","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Jones","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guillermo","family":"Solorzano","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoyan","family":"Li","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zabeen","family":"Mahuwala","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vishakhadatta (Vish) Mathur","family":"Kumaraswamy","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Colin","family":"Quinn","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Baer","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Borg","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karthikeyan","family":"Bhuvaneswaran","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jasdeep","family":"Kaur","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sam","family":"Maiser","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Seward B.","family":"Rutkove","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew","family":"Mundwiler","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jenny A.","family":"Meyer","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pooja","family":"Rao","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raymond","family":"Roos","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ali A.","family":"Habib","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tahseen","family":"Mozaffar","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manisha Kak","family":"Korb","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Mullen","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathaniel M","family":"Robbins","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathan","family":"Carberry","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Volkan","family":"Granit","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raghav","family":"Govindarajan","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina N.","family":"Fournier","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bjorn","family":"Oskarsson","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leila","family":"Darki","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rodrigo","family":"Rodriguez","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miguel","family":"Chuguilin","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Whitney","family":"McNeely","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Montserrat","family":"Diaz-Abad","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter H.","family":"Jin","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chandana","family":"Chauhan","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"Bobenhouse","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathan P.","family":"Staff","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ghazala","family":"Hayat","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luisa","family":"Arroyave","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Abbey","family":"Bailey","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesse","family":"Bailey","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Victoria","family":"Barlow","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Allison","family":"Bulat","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Genevive","family":"Changkuon","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa","family":"Cirino","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Deignan","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma","family":"Deirmendjian","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annette","family":"De Mattos","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sofia","family":"DiStefano","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristin","family":"Drake","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michaela","family":"Estes","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kenneth","family":"Faulconer","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Precious","family":"Figueroa-Szostek","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tessa","family":"Garozzo","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Meredith Gibbons","family":"Hasenoehrl","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Henrique","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalie","family":"Henrique","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Samuel","family":"Hurwitz","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Courtney","family":"Igne","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liam","family":"Irwin","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katie","family":"Jentoft","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Boglarka","family":"Jordan","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Igor","family":"Katsovskiy","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olga","family":"Kharakozova","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Taylor","family":"Kolvek","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander","family":"Korin","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thuong","family":"La","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Haining","family":"Li","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joey","family":"Nguyen","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilya","family":"Novak","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ricardo","family":"Ortiz","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joe","family":"Ostrow","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaclyn","family":"Pagliaro","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jack","family":"Palillo","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Payal","family":"Patel","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janae","family":"Patterson","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Minh","family":"Phan","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Najla","family":"Popel","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Serena","family":"Proueng","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesse","family":"Rosenthal","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Small","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalia","family":"Tarasenko","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mirna","family":"Thomas","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Prasha","family":"Vigneswaran","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yusra","family":"Wahab","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isaac","family":"Whitworth","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Spencer","family":"Wright","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariah","family":"Connolly","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana","family":"De Santiago","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adrian","family":"Felix","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karly","family":"Garrett","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jenny","family":"Hamilton","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kamran","family":"Khan","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marlee","family":"Lovett","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda","family":"Nelson","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marissa","family":"Pabon","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana","family":"Rede","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick","family":"Bolger","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ahmed","family":"Fetouh","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joan","family":"Woodcook","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cornelia","family":"Kamp","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julie","family":"Kennedy","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew","family":"McGarry","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Margherita","family":"Torti","sequence":"additional","affiliation":[{"name":"Writing Committee for the HEALEY ALS Platform Trial"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James D.","family":"Berry","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicholas J.","family":"Maragakis","sequence":"additional","affiliation":[{"name":"Johns Hopkins University, Baltimore, Maryland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric A.","family":"Macklin","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lori B.","family":"Chibnik","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melanie","family":"Quintana","sequence":"additional","affiliation":[{"name":"Berry Consultants, Austin, Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin R.","family":"Saville","sequence":"additional","affiliation":[{"name":"Berry Consultants, Austin, Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michelle A.","family":"Detry","sequence":"additional","affiliation":[{"name":"Berry Consultants, Austin, Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matteo","family":"Vestrucci","sequence":"additional","affiliation":[{"name":"Berry Consultants, Austin, Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joseph","family":"Marion","sequence":"additional","affiliation":[{"name":"Berry Consultants, Austin, Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"McGlothlin","sequence":"additional","affiliation":[{"name":"Berry Consultants, Austin, Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elijah W.","family":"Stommel","sequence":"additional","affiliation":[{"name":"Dartmouth Hitchcock Medical Center, Lebanon, New Hampshire"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marianne","family":"Chase","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lindsay","family":"Pothier","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brittney A.","family":"Harkey","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hong","family":"Yu","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alex","family":"Sherman","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeremy","family":"Shefner","sequence":"additional","affiliation":[{"name":"Barrow Neurological Institute, Phoenix, Arizona"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Meghan","family":"Hall","sequence":"additional","affiliation":[{"name":"Barrow Neurological Institute, Phoenix, Arizona"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gale","family":"Kittle","sequence":"additional","affiliation":[{"name":"Barrow Neurological Institute, Phoenix, Arizona"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suma","family":"Babu","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jinsy","family":"Andrews","sequence":"additional","affiliation":[{"name":"Columbia University Medical Center, New York, New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Derek","family":"D\u2019Agostino","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Tustison","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erica","family":"Scirocco","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elisa","family":"Giacomelli","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gustavo","family":"Alameda","sequence":"additional","affiliation":[{"name":"Holy Cross Hospital, Fort Lauderdale, Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eduardo","family":"Locatelli","sequence":"additional","affiliation":[{"name":"Holy Cross Hospital, Fort Lauderdale, Florida"},{"name":"Nova Southeastern University, Fort Lauderdale, Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Doreen","family":"Ho","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adam","family":"Quick","sequence":"additional","affiliation":[{"name":"Ohio State University, Columbus"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Senda","family":"Ajroud-Driss","sequence":"additional","affiliation":[{"name":"Northwestern University, Chicago, Illinois"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"Katz","sequence":"additional","affiliation":[{"name":"California Pacific Medical Center, San Francisco"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daragh","family":"Heitzman","sequence":"additional","affiliation":[{"name":"Texas Neurology, Dallas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stanley H.","family":"Appel","sequence":"additional","affiliation":[{"name":"Houston Methodist Neurological Institute, Houston, Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sheetal","family":"Shroff","sequence":"additional","affiliation":[{"name":"Houston Methodist Neurological Institute, Houston, Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kevin J.","family":"Felice","sequence":"additional","affiliation":[{"name":"Hospital for Special Care, New Britain, Connecticut"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zachary","family":"Simmons","sequence":"additional","affiliation":[{"name":"Penn State Milton S. Hershey Medical Center, Hershey, Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Timothy","family":"Miller","sequence":"additional","affiliation":[{"name":"Washington University in St Louis, St Louis, Missouri"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicholas","family":"Olney","sequence":"additional","affiliation":[{"name":"Providence ALS Clinic, Portland, Oregon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael D.","family":"Weiss","sequence":"additional","affiliation":[{"name":"University of Washington, Seattle"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen A.","family":"Goutman","sequence":"additional","affiliation":[{"name":"University of Michigan, Ann Arbor"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"Jr","given":"Joseph Americo","family":"Fernandes","sequence":"additional","affiliation":[{"name":"University of Nebraska Medical Center, Omaha"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Omar","family":"Jawdat","sequence":"additional","affiliation":[{"name":"University of Kansas Medical Center, Kansas City"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Margaret Ayo","family":"Owegi","sequence":"additional","affiliation":[{"name":"University of Massachusetts, Worcester"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Foster","sequence":"additional","affiliation":[{"name":"University of Colorado Anschutz, Denver"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tuan","family":"Vu","sequence":"additional","affiliation":[{"name":"University of South Florida, Morsani College of Medicine, Tampa"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hristelina","family":"Ilieva","sequence":"additional","affiliation":[{"name":"Jefferson Weinberg ALS Center, Philadelphia, Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel S.","family":"Newman","sequence":"additional","affiliation":[{"name":"Henry Ford Hospital, Detroit, Michigan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ximena","family":"Arcila-Londono","sequence":"additional","affiliation":[{"name":"Henry Ford Hospital, Detroit, Michigan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carlayne","family":"Jackson","sequence":"additional","affiliation":[{"name":"University of Texas Health Science Center, San Antonio"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shafeeq","family":"Ladha","sequence":"additional","affiliation":[{"name":"Barrow Neurological Institute, Phoenix, Arizona"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Terry","family":"Heiman-Patterson","sequence":"additional","affiliation":[{"name":"Temple University, Philadelphia, Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"Caress","sequence":"additional","affiliation":[{"name":"Wake Forest University School of Medicine, Winston-Salem, North Carolina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Swenson","sequence":"additional","affiliation":[{"name":"University of Iowa, Iowa City"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amanda","family":"Peltier","sequence":"additional","affiliation":[{"name":"Vanderbilt University Medical Center, Nashville, Tennessee"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Lewis","sequence":"additional","affiliation":[{"name":"Cedars-Sinai Medical Center, Los Angeles, California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dominic","family":"Fee","sequence":"additional","affiliation":[{"name":"Medical College of Wisconsin, Milwaukee"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Elliott","sequence":"additional","affiliation":[{"name":"University of Virginia, Charlottesville"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Bedlack","sequence":"additional","affiliation":[{"name":"Duke University, Durham, North Carolina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edward J.","family":"Kasarskis","sequence":"additional","affiliation":[{"name":"University of Kentucky, Lexington"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lauren","family":"Elman","sequence":"additional","affiliation":[{"name":"University of Pennsylvania, Philadelphia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Rosenfeld","sequence":"additional","affiliation":[{"name":"Loma Linda University School of Medicine, Loma Linda, California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Walk","sequence":"additional","affiliation":[{"name":"University of Minnesota Twin Cities ALS Research Consortium, Minneapolis"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Courtney E.","family":"McIlduff","sequence":"additional","affiliation":[{"name":"Beth Israel Deaconess Medical Center, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Twydell","sequence":"additional","affiliation":[{"name":"Spectrum Health Medical Group, Grand Rapids, Michigan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eufrosina","family":"Young","sequence":"additional","affiliation":[{"name":"SUNY Upstate, Syracuse, New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristin","family":"Johnson","sequence":"additional","affiliation":[{"name":"Ochsner Health System, Covington, Louisiana"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kourosh","family":"Rezania","sequence":"additional","affiliation":[{"name":"University of Chicago, Chicago, Illinois"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Namita A.","family":"Goyal","sequence":"additional","affiliation":[{"name":"University of California, Irvine Medical Center, Irvine"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey A.","family":"Cohen","sequence":"additional","affiliation":[{"name":"Dartmouth Hitchcock Medical Center, Lebanon, New Hampshire"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Benatar","sequence":"additional","affiliation":[{"name":"University of Miami, Miami, Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vovanti","family":"Jones","sequence":"additional","affiliation":[{"name":"University of Missouri, Columbia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"Glass","sequence":"additional","affiliation":[{"name":"Emory University, Atlanta, Georgia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaimin","family":"Shah","sequence":"additional","affiliation":[{"name":"Mayo Clinic Jacksonville, Jacksonville, Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Said R.","family":"Beydoun","sequence":"additional","affiliation":[{"name":"University of Southern California, Los Angeles"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James P.","family":"Wymer","sequence":"additional","affiliation":[{"name":"University of Florida Gainesville"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lindsay","family":"Zilliox","sequence":"additional","affiliation":[{"name":"University of Maryland School of Medicine, Baltimore"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shakti","family":"Nayar","sequence":"additional","affiliation":[{"name":"Georgetown University, Washington, DC"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gary L.","family":"Pattee","sequence":"additional","affiliation":[{"name":"Neurology Associates, Lincoln, Nebraska"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Martinez-Thompson","sequence":"additional","affiliation":[{"name":"Mayo Clinic, Rochester, Minnesota"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Austin","family":"Rynders","sequence":"additional","affiliation":[{"name":"Neurology Associates, Lincoln, Nebraska"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacob","family":"Evan","sequence":"additional","affiliation":[{"name":"Neurology Associates, Lincoln, Nebraska"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeremy","family":"Evan","sequence":"additional","affiliation":[{"name":"Neurology Associates, Lincoln, Nebraska"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alan","family":"Hartford","sequence":"additional","affiliation":[{"name":"Clene Nanomedicine, North East, Maryland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marjan","family":"Sepassi","sequence":"additional","affiliation":[{"name":"Clene Nanomedicine, North East, Maryland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karen S.","family":"Ho","sequence":"additional","affiliation":[{"name":"Clene Nanomedicine, North East, Maryland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Glanzman","sequence":"additional","affiliation":[{"name":"Clene Nanomedicine, North East, Maryland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin","family":"Greenberg","sequence":"additional","affiliation":[{"name":"Clene Nanomedicine, North East, Maryland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael T.","family":"Hotchkin","sequence":"additional","affiliation":[{"name":"Clene Nanomedicine, North East, Maryland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabrina","family":"Paganoni","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"},{"name":"Spaulding Rehabilitation Hospital, Harvard Medical School, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Merit E.","family":"Cudkowicz","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"HEALEY ALS Platform Trial Study Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"10","reference":[{"issue":"1","key":"joi240158r1","doi-asserted-by":"publisher","first-page":"75","DOI":"10.1016\/S1474-4422(10)70224-6","article-title":"Energy metabolism in amyotrophic lateral sclerosis.","volume":"10","author":"Dupuis","year":"2011","journal-title":"Lancet Neurol"},{"issue":"5-6","key":"joi240158r2","doi-asserted-by":"publisher","first-page":"382","DOI":"10.1159\/000446502","article-title":"Altered metabolic homeostasis in amyotrophic lateral sclerosis: mechanisms of energy imbalance and contribution to disease progression.","volume":"16","author":"Ioannides","year":"2016","journal-title":"Neurodegener Dis"},{"issue":"4","key":"joi240158r3","doi-asserted-by":"publisher","first-page":"489","DOI":"10.1007\/s00401-018-1835-x","article-title":"Energy metabolism in ALS: an underappreciated opportunity?","volume":"135","author":"Vandoorne","year":"2018","journal-title":"Acta Neuropathol"},{"issue":"1","key":"joi240158r4","doi-asserted-by":"publisher","first-page":"1936","DOI":"10.1038\/s41598-020-58709-w","article-title":"Nanocatalytic activity of clean-surfaced, faceted nanocrystalline gold enhances remyelination in animal models of multiple sclerosis.","volume":"10","author":"Robinson","year":"2020","journal-title":"Sci Rep"},{"issue":"8","key":"joi240158r5","doi-asserted-by":"publisher","DOI":"10.1002\/smll.202304082","article-title":"A mechanism underpinning the bioenergetic metabolism-regulating function of gold nanocatalysts.","volume":"20","author":"Wang","year":"2024","journal-title":"Small"},{"issue":"20","key":"joi240158r6","doi-asserted-by":"publisher","first-page":"10836","DOI":"10.1074\/jbc.M115.698779","article-title":"Enhancing NAD+ salvage pathway reverts the toxicity of primary astrocytes expressing amyotrophic lateral sclerosis-linked mutant superoxide dismutase 1 (SOD1).","volume":"291","author":"Harlan","year":"2016","journal-title":"J Biol Chem"},{"key":"joi240158r7","doi-asserted-by":"publisher","DOI":"10.1016\/j.expneurol.2020.113219","article-title":"Evaluation of the NAD+ biosynthetic pathway in ALS patients and effect of modulating NAD+ levels in hSOD1-linked ALS mouse models.","volume":"327","author":"Harlan","year":"2020","journal-title":"Exp Neurol"},{"issue":"2","key":"joi240158r8","doi-asserted-by":"publisher","first-page":"284","DOI":"10.7150\/ijbs.38487","article-title":"Nicotinamide riboside enhances mitochondrial proteostasis and adult neurogenesis through activation of mitochondrial unfolded protein response signaling in the brain of ALS SOD1G93A mice.","volume":"16","author":"Zhou","year":"2020","journal-title":"Int J Biol Sci"},{"issue":"1","key":"joi240158r9","doi-asserted-by":"publisher","first-page":"4287","DOI":"10.1038\/s41598-022-08068-5","article-title":"Mitochondrial dysregulation occurs early in ALS motor cortex with TDP-43 pathology and suggests maintaining NAD+ balance as a therapeutic strategy.","volume":"12","author":"Gautam","year":"2022","journal-title":"Sci Rep"},{"issue":"4","key":"joi240158r10","doi-asserted-by":"publisher","first-page":"1379","DOI":"10.1038\/s41418-020-00664-0","article-title":"ALS motor neurons exhibit hallmark metabolic defects that are rescued by SIRT3 activation.","volume":"28","author":"Hor","year":"2021","journal-title":"Cell Death Differ"},{"issue":"1","key":"joi240158r11","doi-asserted-by":"crossref","DOI":"10.1136\/bmjopen-2020-041479","article-title":"Study protocol of RESCUE-ALS: a phase 2, randomised, double-blind, placebo-controlled study in early symptomatic amyotrophic lateral sclerosis patients to assess bioenergetic catalysis with CNM-Au8 as a mechanism to slow disease progression.","volume":"11","author":"Vucic","year":"2021","journal-title":"BMJ Open"},{"key":"joi240158r12","doi-asserted-by":"crossref","DOI":"10.1016\/j.eclinm.2023.102036","article-title":"Efficacy and safety of CNM-Au8 in amyotrophic lateral sclerosis (RESCUE-ALS study): a phase 2, randomised, double-blind, placebo-controlled trial and open label extension.","volume":"60","author":"Vucic","year":"2023","journal-title":"EClinicalMedicine"},{"issue":"2","key":"joi240158r13","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1002\/ana.26285","article-title":"Adaptive platform trials to transform amyotrophic lateral sclerosis therapy development.","volume":"91","author":"Paganoni","year":"2022","journal-title":"Ann Neurol"},{"issue":"3","key":"joi240158r14","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1002\/ana.26714","article-title":"Design and statistical innovations in a platform trial for ALS.","volume":"94","author":"Quintana","year":"2023","journal-title":"Ann Neurol"},{"issue":"11","key":"joi240158r15","doi-asserted-by":"crossref","first-page":"1341","DOI":"10.1002\/(SICI)1097-0258(19990615)18:11<1341::AID-SIM129>3.0.CO;2-7","article-title":"Combining mortality and longitudinal measures in clinical trials.","volume":"18","author":"Finkelstein","year":"1999","journal-title":"Stat Med"},{"issue":"3","key":"joi240158r16","doi-asserted-by":"crossref","first-page":"162","DOI":"10.3109\/21678421.2012.762930","article-title":"The Combined Assessment of Function and Survival (CAFS): a new endpoint for ALS clinical trials.","volume":"14","author":"Berry","year":"2013","journal-title":"Amyotroph Lateral Scler Frontotemporal Degener"},{"issue":"6","key":"joi240158r17","doi-asserted-by":"crossref","first-page":"1324","DOI":"10.1183\/09031936.00080312","article-title":"Multi-ethnic reference values for spirometry for the 3-95-yr age range: the global lung function 2012 equations.","volume":"40","author":"Quanjer","year":"2012","journal-title":"Eur Respir J"},{"issue":"7","key":"joi240158r18","doi-asserted-by":"crossref","first-page":"1103","DOI":"10.1002\/acn3.51078","article-title":"Longitudinal biomarkers in amyotrophic lateral sclerosis.","volume":"7","author":"Huang","year":"2020","journal-title":"Ann Clin Transl Neurol"},{"issue":"1","key":"joi240158r19","doi-asserted-by":"crossref","first-page":"e59","DOI":"10.1212\/WNL.0000000000009559","article-title":"Validation of serum neurofilaments as prognostic and potential pharmacodynamic biomarkers for ALS.","volume":"95","author":"Benatar","year":"2020","journal-title":"Neurology"},{"issue":"12","key":"joi240158r20","doi-asserted-by":"crossref","first-page":"1137","DOI":"10.1212\/WNL.0000000000003741","article-title":"Urinary p75ECD: a prognostic, disease progression, and pharmacodynamic biomarker in ALS.","volume":"88","author":"Shepheard","year":"2017","journal-title":"Neurology"},{"issue":"1","key":"joi240158r21","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1136\/jnnp-2013-306865","article-title":"Use of clinical staging in amyotrophic lateral sclerosis for phase 3 clinical trials.","volume":"86","author":"Balendra","year":"2015","journal-title":"J Neurol Neurosurg Psychiatry"},{"issue":"12","key":"joi240158r22","doi-asserted-by":"crossref","first-page":"1099","DOI":"10.1056\/NEJMoa2204705","article-title":"Trial of antisense oligonucleotide tofersen for SOD1 ALS.","volume":"387","author":"Miller","year":"2022","journal-title":"N Engl J Med"}],"container-title":["JAMA"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/jamanetwork.com\/journals\/jama\/articlepdf\/2830508\/jama_berry_2025_oi_240158_1742927563.7361.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,4,4]],"date-time":"2025-04-04T01:32:47Z","timestamp":1743730367000},"score":1,"resource":{"primary":{"URL":"https:\/\/jamanetwork.com\/journals\/jama\/fullarticle\/2830508"}},"subtitle":["The HEALEY ALS Platform Trial"],"short-title":[],"issued":{"date-parts":[[2025,4,1]]},"references-count":22,"journal-issue":{"issue":"13","published-print":{"date-parts":[[2025,4,1]]}},"URL":"https:\/\/doi.org\/10.1001\/jama.2024.27643","relation":{},"ISSN":["0098-7484"],"issn-type":[{"value":"0098-7484","type":"print"}],"subject":[],"published":{"date-parts":[[2025,4,1]]}}}