Long-term Safety and Efficacy in Patients with DMD 4 Years Post-Treatment with Delandistrogene Moxeparvovec (SRP-9001) in a Phase 1/2a Study (P3-8.006)

Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques

“…This is a feasible dose level that has been used in rAAV-based gene therapies for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy, which are now both FDA approved. 34 , 35 …”