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Translation of data from preclinical studies to justify any clinical trial must be carefully pondered in order to minimize the risk of clinical trial withdrawal or failure. A rigorous distinction between proof-of-concept and preclinical efficacy studies using animal models is key to support the rationale of a clinical trial involving patients. This Review evaluates the experience accumulated by the TREAT-NMD Advisory Committee for Therapeutics, which provides detailed constructive feedback on clinical proposals for neuromuscular diseases submitted by researchers in both academia and industry, and emphasizes that a timely critical review of preclinical efficacy data from animal models, including biomarkers for specific diseases, combined with adherence to existing guidelines and standard protocols, can significantly help to de-risk clinical programs and prevent disappointments and costly engagement.<\/jats:p>","DOI":"10.1242\/dmm.042903","type":"journal-article","created":{"date-parts":[[2020,2,7]],"date-time":"2020-02-07T14:34:15Z","timestamp":1581086055000},"source":"Crossref","is-referenced-by-count":27,"title":["Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)"],"prefix":"10.1242","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5949-0900","authenticated-orcid":false,"given":"Raffaella","family":"Willmann","sequence":"first","affiliation":[{"name":"Swiss Foundation for Research on Muscle Diseases 1 , 2016 Cortaillod , Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanne","family":"Lee","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Centre for Life, Newcastle University and Newcastle Hospitals NHS Foundation Trust 2 , Newcastle upon Tyne NE7 7DN , UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cathy","family":"Turner","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Centre for Life, Newcastle University and Newcastle Hospitals NHS Foundation Trust 2 , Newcastle upon Tyne NE7 7DN , UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kanneboyina","family":"Nagaraju","sequence":"additional","affiliation":[{"name":"School of Pharmacy and Pharmaceutical Sciences, Binghamton University 3 , New York , NY 13902-6000, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annemieke","family":"Aartsma-Rus","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Centre for Life, Newcastle University and Newcastle Hospitals NHS Foundation Trust 2 , Newcastle upon Tyne NE7 7DN , UK"},{"name":"Department of Human Genetics, Leiden University Medical Center 4 , Leiden, 2300 RC , the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dominic J.","family":"Wells","sequence":"additional","affiliation":[{"name":"Neuromuscular Disease Group, Royal Veterinary College 5 , London NW1 0TU , UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn R.","family":"Wagner","sequence":"additional","affiliation":[{"name":"Center for Genetic Muscle Disorders, Kennedy Krieger Institute and the Departments of Neurology and Neuroscience, Johns Hopkins School of Medicine 6 , Baltimore, MD 21205 , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Csimma","sequence":"additional","affiliation":[{"name":"Csimma LLC 7 , Lincoln, MA 01773 , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Volker","family":"Straub","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Centre for Life, Newcastle University and Newcastle Hospitals NHS Foundation Trust 2 , Newcastle upon Tyne NE7 7DN , UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miranda D.","family":"Grounds","sequence":"additional","affiliation":[{"name":"School of Human Sciences, The University of Western Australia 8 , Perth, WA 6009 , Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5652-7341","authenticated-orcid":false,"given":"Annamaria","family":"De Luca","sequence":"additional","affiliation":[{"name":"University of Bari Aldo Moro 9 Unit of Pharmacology, Department of Pharmacy and Drug Sciences , , 70125 Bari , Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"237","published-online":{"date-parts":[[2020,2,7]]},"reference":[{"key":"2024061302411846000_DMM042903C1","doi-asserted-by":"publisher","first-page":"39","DOI":"10.1007\/s00395-018-0696-8","article-title":"Practical guidelines for rigor and reproducibility in preclinical and clinical studies on cardioprotection","volume":"113","author":"B\u00f8tker","year":"2018","journal-title":"Basic Res. 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