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

• Main Authors: Raffaella Willmann (Author), Joanne Lee (Author), Cathy Turner (Author), Kanneboyina Nagaraju (Author), Annemieke Aartsma-Rus (Author), Dominic J. Wells (Author), Kathryn R. Wagner (Author), Cristina Csimma (Author), Volker Straub (Author), Miranda D. Grounds (Author), Annamaria De Luca (Author), James Dowling (Author), Maaike van Putten (Author)
• Format: Book
• Published: The Company of Biologists, 2020-02-01T00:00:00Z.
• Subjects: article
• Online Access: 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...