{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,12]],"date-time":"2025-11-12T20:56:13Z","timestamp":1762980973854,"version":"3.40.5"},"reference-count":24,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2020,8,13]],"date-time":"2020-08-13T00:00:00Z","timestamp":1597276800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2020,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Inotersen, an antisense oligonucleotide inhibitor of transthyretin (TTR) protein production, demonstrated significant benefit versus placebo in the modified Neuropathy Impairment Score (NIS)\u2009+7 neurophysiologic tests (mNIS+7) in patients with hereditary TTR\u2010mediated amyloidosis (hATTR) with polyneuropathy. This analysis assessed the mNIS+7 components by anatomic location and the lower limb function (LLF) test.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Adults with hATTR in the NEURO\u2010TTR trial (NCT01737398) were randomly assigned to receive weekly doses of subcutaneous inotersen 300\u2009mg or placebo for 65\u2009weeks. The mNIS+7 and LLF were assessed at 35 and 66\u2009weeks.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>All major mNIS+7 components (muscle weakness, muscle stretch reflexes, sensation) and the LLF showed significant efficacy in patients receiving inotersen versus placebo; however, NIS\u2010reflexes (upper limb), touch pressure (upper and lower limbs), and heart rate during deep breathing did not show significant effects.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>The results of this analysis reinforce the beneficial effect of inotersen on slowing neuropathy progression in patients with hATTR polyneuropathy.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.27022","type":"journal-article","created":{"date-parts":[[2020,7,12]],"date-time":"2020-07-12T11:55:30Z","timestamp":1594554930000},"page":"502-508","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["<scp>mNIS<\/scp>+7 <scp>and lower limb function in inotersen treatment of<\/scp> hereditary transthyretin\u2010mediated amyloidosis"],"prefix":"10.1002","volume":"62","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0212-8254","authenticated-orcid":false,"given":"P. James B.","family":"Dyck","sequence":"first","affiliation":[{"name":"Department of Neurology Mayo Clinic  Rochester Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"John C.","family":"Kincaid","sequence":"additional","affiliation":[{"name":"Department of Neurology Indiana University  Indianapolis Indiana USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Janice F.","family":"Wiesman","sequence":"additional","affiliation":[{"name":"Department of Neurology New York University  New York New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael","family":"Polydefkis","sequence":"additional","affiliation":[{"name":"Department of Neurology Johns Hopkins University  Baltimore Maryland USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"William J.","family":"Litchy","sequence":"additional","affiliation":[{"name":"Department of Neurology Mayo Clinic  Rochester Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michelle L.","family":"Mauermann","sequence":"additional","affiliation":[{"name":"Department of Neurology Mayo Clinic  Rochester Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elizabeth J.","family":"Ackermann","sequence":"additional","affiliation":[{"name":"Otonomy, Inc.  San Diego California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Spencer","family":"Guthrie","sequence":"additional","affiliation":[{"name":"Aurora Bio  San Francisco California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael","family":"Pollock","sequence":"additional","affiliation":[{"name":"Akcea Therapeutics, Inc.  Cambridge Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shiangtung W.","family":"Jung","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Inc.  Carlsbad California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brenda F.","family":"Baker","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Inc.  Carlsbad California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Peter J.","family":"Dyck","sequence":"additional","affiliation":[{"name":"Department of Neurology Mayo Clinic  Rochester Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2020,8,13]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/jns.12153"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.20821"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1185\/03007995.2012.754348"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1093\/eurheartj\/ehr383"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.3109\/13506129609014354"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1093\/eurheartj\/ehs123"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1716793"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1716153"},{"issue":"7","key":"e_1_2_10_10_1","first-page":"S107","article-title":"Hereditary ATTR amyloidosis: burden of illness and diagnostic challenges","volume":"23","author":"Gertz MA","year":"2017","journal-title":"Am J Manag Care"},{"key":"e_1_2_10_11_1","unstructured":"TEGSEDI (inotersen) injection for subcutaneous use [prescribing information].Boston MA:Akcea Therapeutics;2018."},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1080\/13506129.2016.1191458"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.25563"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1017\/S0317167100002043"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2014.06.041"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2019.116424"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.410380323"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2013.283815"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2018.08.031"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.43.8.1508"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21661"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneurol.2012.1481"},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23982"},{"key":"e_1_2_10_24_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24243"},{"key":"e_1_2_10_25_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23765"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fmus.27022","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27022","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.27022","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27022","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,5]],"date-time":"2023-09-05T16:44:36Z","timestamp":1693932276000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.27022"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,8,13]]},"references-count":24,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2020,10]]}},"alternative-id":["10.1002\/mus.27022"],"URL":"https:\/\/doi.org\/10.1002\/mus.27022","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"type":"print","value":"0148-639X"},{"type":"electronic","value":"1097-4598"}],"subject":[],"published":{"date-parts":[[2020,8,13]]},"assertion":[{"value":"2019-11-04","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-07-07","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-08-13","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}