{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T09:31:27Z","timestamp":1793266287156,"version":"4.5.0"},"reference-count":24,"publisher":"SAGE Publications","issue":"6","license":[{"start":{"date-parts":[[2026,4,24]],"date-time":"2026-04-24T00:00:00Z","timestamp":1776988800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,4,24]],"date-time":"2026-04-24T00:00:00Z","timestamp":1776988800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2026,11]]},"abstract":"<jats:p>The TREAT-NMD Advisory Committee for Therapeutics (TACT) provides academic and industry applicants involved in therapy development in the neuromuscular disease (NMD) space with constructive, multidisciplinary expert advice in order to help derisk NMD drug development and avoid potential therapies entering clinical trials prematurely and to improve trial design. The ultimate goal of TACT is to reduce the risk of type 1 errors (concluding a treatment is effective, while it is not), type 2 errors (concluding a treatment is not effective, while it is), or risks of questions and regulatory rejections later on in development.<\/jats:p>\n                  <jats:p>\n                    TACT celebrated its 15\n                    <jats:sup>th<\/jats:sup>\n                    birthday in 2024. From 2009\u20132024 TACT reviewed 90 applications, 68 from Industry and 22 from Academia. This perspective paper summarizes how TACT has evolved from its initiation. Furthermore, based on our experience, we list recurrent mistakes, such as not collecting sufficient information in pre-clinical studies, omitting wild type references in animal studies and choosing the wrong or too many outcome measures in clinical trials. By sharing common errors in preclinical and clinical trial design and providing lessons learned we aim to further improve therapy development in the NMD space.\n                  <\/jats:p>","DOI":"10.1177\/22143602261438534","type":"journal-article","created":{"date-parts":[[2026,4,24]],"date-time":"2026-04-24T14:41:47Z","timestamp":1777041707000},"page":"1215-1227","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":1,"title":["TREAT-NMD advisory committee for therapeutics: Preclinical and clinical learnings from 15 years of TACT"],"prefix":"10.1177","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1565-654X","authenticated-orcid":false,"given":"Annemieke","family":"Aartsma-Rus","sequence":"first","affiliation":[{"name":"Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Robertson","sequence":"additional","affiliation":[{"name":"TREAT-NMD, Newcastle upon Tyne, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lindsay N.","family":"Alfano","sequence":"additional","affiliation":[{"name":"Nationwide Children's Hospital, Columbus OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Mayhew","sequence":"additional","affiliation":[{"name":"The John Walton Muscular Dystrophy Research Centre"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather","family":"Gordish-Dressman","sequence":"additional","affiliation":[{"name":"Childrens National Hospital"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mike","family":"Kelly","sequence":"additional","affiliation":[{"name":"CureDuchenne, Newport Beach, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carl","family":"Morris","sequence":"additional","affiliation":[{"name":"PHDLConsulting, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Volker","family":"Straub","sequence":"additional","affiliation":[{"name":"The John Walton Muscular Dystrophy Research Centre"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Conrad C.","family":"Weihl","sequence":"additional","affiliation":[{"name":"Department of Neurology, Washington University School of Medicine, Saint Louis, Misssouri, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Colin","family":"Werth","sequence":"additional","affiliation":[{"name":"Parent project muscular dystrophy adult advisory committee (PAAC)"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6665-7887","authenticated-orcid":false,"given":"Olivier M.","family":"Dorchies","sequence":"additional","affiliation":[{"name":"University of Grenoble-Alpes, Laboratory of Fundamental and Applied Bionergetics (Inserm U1055)"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0004-8204-2980","authenticated-orcid":false,"given":"Amanda","family":"Riviere","sequence":"additional","affiliation":[{"name":"Genethon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kanneboyina","family":"Nagaraju","sequence":"additional","affiliation":[{"name":"Binghamton University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5315-0278","authenticated-orcid":false,"given":"Kathryn R.","family":"Wagner","sequence":"additional","affiliation":[{"name":"TREAT-NMD, Newcastle upon Tyne, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,4,24]]},"reference":[{"key":"e_1_3_2_2_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-015-0258-1"},{"key":"e_1_3_2_3_2","doi-asserted-by":"publisher","DOI":"10.1038\/d41573-019-00199-1"},{"key":"e_1_3_2_4_2","doi-asserted-by":"publisher","DOI":"10.1242\/dmm.042903"},{"key":"e_1_3_2_5_2","doi-asserted-by":"publisher","DOI":"10.1002\/mds.29032"},{"key":"e_1_3_2_6_2","doi-asserted-by":"publisher","DOI":"10.1177\/22143602251399511"},{"key":"e_1_3_2_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(16)30035-7"},{"key":"e_1_3_2_8_2","doi-asserted-by":"publisher","DOI":"10.1038\/533452a"},{"key":"e_1_3_2_9_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.05.003"},{"key":"e_1_3_2_10_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-170288"},{"key":"e_1_3_2_11_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2011.04.012"},{"key":"e_1_3_2_12_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-180324"},{"key":"e_1_3_2_13_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2010.09.014"},{"key":"e_1_3_2_14_2","doi-asserted-by":"publisher","DOI":"10.1038\/nature11556"},{"key":"e_1_3_2_15_2","doi-asserted-by":"publisher","DOI":"10.1096\/fj.201802488R"},{"key":"e_1_3_2_16_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0182704"},{"key":"e_1_3_2_17_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(23)00313-7"},{"key":"e_1_3_2_18_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0304099"},{"key":"e_1_3_2_19_2","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2023.0027"},{"key":"e_1_3_2_20_2","first-page":"55","volume-title":"Good 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General principles of preclinical study design. In: Bespalov A, MC M, Steckler T (eds) Good research practice in non-clinical pharmacology and biomedicine. 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