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
  • DYN101-C101 / 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





Intervention AT132 DYN101
Principal investigators
Andreea Seferian Anthony Béhin
Sponsor Audentes Dynacure
Fundings Audentes
Study status Ongoing On hold
Recruitment status Closed Closed
Population Child Adult
  + infos on  + infos on