- 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 |