Myotubular Myopathy

  • ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients
  • Unite-CNM: A Phase 1/2 trial on the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of DYN101 in patients ≥ 16 years of age with centronuclear myopathies caused by mutations in DNM2 or MTM1
  • Dynamic: A phase 1/2, multicenter, open-label, dose-confirming study to evaluate the safety and preliminary efficacy of DYN101 in children aged 2 to 17 with centronuclear myopathy caused by mutations in MTM1 or DNM2

 

Acronym

ASPIRO

Unite-CNM

Dynamic

Intervention AT132 DYN101 DYN101
Principal investigators
Andreea Seferian Anthony Béhin Emmanuelle Lagrue
Sponsor Audentes Dynacure Dynacure
Fundings Audentes
Study status Ongoing Ongoing In préparation
Recruitment status Closed Ongoing In préparation
Population Child Adult Child
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