A Phase 1/2, multicenter, open-label, dose-confirmation trial to evaluate the safety and preliminary efficacy of DYN101 in subjects 2 to 17 years of age with centronuclear myopathy caused by mutations in MTM1 or DNM2. - Research Using an Investigational Treatment for CNM (DyNaMic) A Phase 1/2, multicenter, open-label, dose-confirmation trial to evaluate the safety and preliminary ...
Centronuclear myopathy (CNM), in subjects 2 to 17 years of age (all ages inclusive)caused by mutations in the myotubularin1 (MTM1) or dynamin 2 (DNM2) gene MedDRA version: 20.0;Level: HLT;Classification code 10028640;Term: Myopathies;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Centronuclear myopathy (CNM), in subjects 2 to 17 years of age (all ages inclusive)caused by mutatio ...
Product Name: DYN101 Product Code: DYN101 INN or Proposed INN: DYN101