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

 

Acronym

ASPIRO

DYN101-C101
/ UNITE-CNM
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 clinicaltrials.gov  + infos on clinicaltrials.gov