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

摘要: Clinical trials for rare neuromuscular diseases imply, among other investments, a high emotional burden the whole disease community. Translation of data from preclinical studies to justify any clinical trial must be carefully pondered in order minimize risk withdrawal or failure. A rigorous distinction between proof-of-concept and efficacy using animal models is key support rationale involving patients. This Review evaluates experience accumulated by TREAT-NMD Advisory Committee Therapeutics, which provides detailed constructive feedback on proposals submitted researchers both academia industry, emphasizes that timely critical review models, including biomarkers specific diseases, combined with adherence existing guidelines standard protocols, can significantly help de-risk programs prevent disappointments costly engagement.