Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)

• المؤلفون الرئيسيون: Raffaella Willmann (مؤلف), Joanne Lee (مؤلف), Cathy Turner (مؤلف), Kanneboyina Nagaraju (مؤلف), Annemieke Aartsma-Rus (مؤلف), Dominic J. Wells (مؤلف), Kathryn R. Wagner (مؤلف), Cristina Csimma (مؤلف), Volker Straub (مؤلف), Miranda D. Grounds (مؤلف), Annamaria De Luca (مؤلف), James Dowling (مؤلف), Maaike van Putten (مؤلف)
• التنسيق: كتاب
• منشور في: The Company of Biologists, 2020-02-01T00:00:00Z.
• الموضوعات: article
• الوصول للمادة أونلاين: Connect to this object online.

Clinical trials for rare neuromuscular diseases imply, among other investments, a high emotional burden for the whole disease community. 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...