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

 

Acronym

ASPIRO

Intervention AT132
Principal investigators
Andreea Seferian
Sponsor Audentes
Fundings Audentes
Study status Ongoing
Recruitment status Closed
Population Child
  + infos on clinicaltrials.gov