{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T02:50:08Z","timestamp":1791946208616,"version":"4.3.4"},"reference-count":12,"publisher":"Wiley","issue":"9","license":[{"start":{"date-parts":[[2025,9,18]],"date-time":"2025-09-18T00:00:00Z","timestamp":1758153600000},"content-version":"vor","delay-in-days":17,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Euro J of Neurology"],"published-print":{"date-parts":[[2025,9]]},"abstract":"ABSTRACT<\/jats:title>\n \n Introduction\/Aims<\/jats:title>\n \n Sodium phenylbutyrate (\n PB<\/jats:styled-content>\n ) and taurursodiol (\n TURSO<\/jats:styled-content>\n )\u2014\n PB<\/jats:styled-content>\n and\n TURSO<\/jats:styled-content>\n \u2014was approved as a treatment for amyotrophic lateral sclerosis (\n ALS<\/jats:styled-content>\n ) in the United States in 2022 based on the results of a phase 2 trial, but was voluntarily withdrawn from the market in 2024 after negative results from its phase 3 trial. The objective of our study was to describe the real\u2010world clinical experience with\n PB<\/jats:styled-content>\n and\n TURSO<\/jats:styled-content>\n at one\n ALS<\/jats:styled-content>\n clinic.\n <\/jats:p>\n <\/jats:sec>\n \n Methods<\/jats:title>\n \n This was a retrospective chart review of all\n ALS<\/jats:styled-content>\n patients receiving a\n PB<\/jats:styled-content>\n and\n TURSO<\/jats:styled-content>\n prescription at the Massachusetts General Hospital\n ALS<\/jats:styled-content>\n Clinic between October 1, 2022, and September 30, 2023. Data were extracted from patients' electronical medical records up to December 31, 2023.\n <\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n \n A total of 441\n ALS<\/jats:styled-content>\n patients received a\n PB<\/jats:styled-content>\n and\n TURSO<\/jats:styled-content>\n prescription, with 329 (75%) initiating the medication. The average length of treatment was 285\u2009days, and the rate of drug discontinuation during the study period was 41% (N=135). The most common reason for drug discontinuation was side effects (N=93, 69%), with the most common being gastrointestinal issues (N=69). No hospitalizations, deaths, or serious adverse events were considered related to treatment with\n PB<\/jats:styled-content>\n and\n TURSO.<\/jats:styled-content>\n <\/jats:p>\n <\/jats:sec>\n \n Discussion<\/jats:title>\n Experience in real\u2010world clinical settings can help supplement trial data with information on the drug performance at various stages of disease progression. Adverse events impacted treatment persistence in routine clinical practice, underscoring the need for vigilant monitoring and tailored supportive interventions to optimize treatment adherence.<\/jats:p>\n <\/jats:sec>","DOI":"10.1111\/ene.70360","type":"journal-article","created":{"date-parts":[[2025,9,18]],"date-time":"2025-09-18T14:18:17Z","timestamp":1758205097000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Real\u2010World Clinical Experience With Sodium Phenylbutyrate and Taurursodiol at a Single Amyotrophic Lateral Sclerosis Center in the United States"],"prefix":"10.1111","volume":"32","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0615-891X","authenticated-orcid":false,"given":"Narghes","family":"Calcagno","sequence":"first","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School Boston Massachusetts USA"},{"name":"Department of Neurology and Laboratory of Neuroscience IRCCS Istituto Auxologico Italiano Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-9427-1452","authenticated-orcid":false,"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 Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erin","family":"Clampffer","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 Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Grace","family":"Addy","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 Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sean","family":"Morgan","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 Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neel","family":"Parikh","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 Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6964-9842","authenticated-orcid":false,"given":"Dylan V.","family":"Neel","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 Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Scalia","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 Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rebekah","family":"Young","sequence":"additional","affiliation":[{"name":"Amylyx Pharmaceuticals, Inc. Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matteo","family":"Locatelli","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 Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4941-1682","authenticated-orcid":false,"given":"deMauri S.","family":"Mackie","sequence":"additional","affiliation":[{"name":"Amylyx Pharmaceuticals, Inc. Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0898-6081","authenticated-orcid":false,"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 Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0505-1168","authenticated-orcid":false,"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 Massachusetts USA"},{"name":"Spaulding Rehabilitation Hospital Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,9,18]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1603471"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-022-01769-1"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD001447.pub3"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(17)30115-1"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1039\/C7MO00114B"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M111.293282"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1021\/bi026979d"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1916945"},{"key":"e_1_2_10_10_1","unstructured":"L. H.van denBerg \u201cResults From a Global Phase 3 Trial Evaluating an Oral Fixed\u2010Dose Combination of Sodium Phenylbutyrate and Taurursodiol in Amyotrophic Lateral Sclerosis \u201d2024 https:\/\/www.amylyx.com\/document\/final\u2010aan\u2010oral\u2010presentation.pdf."},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28203"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28175"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28096"}],"container-title":["European Journal of Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/ene.70360","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,9,29]],"date-time":"2025-09-29T07:44:06Z","timestamp":1759131846000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/ene.70360"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9]]},"references-count":12,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2025,9]]}},"alternative-id":["10.1111\/ene.70360"],"URL":"https:\/\/doi.org\/10.1111\/ene.70360","archive":["Portico"],"relation":{},"ISSN":["1351-5101","1468-1331"],"issn-type":[{"value":"1351-5101","type":"print"},{"value":"1468-1331","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,9]]},"assertion":[{"value":"2025-09-06","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-09-18","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e70360"}}