Search Results for: microdystrophin

AAV-Mediated Therapies

Gene therapy is a promising treatment option for genetic conditions such as muscular dystrophy. It involves correcting the genetic defect by introducing a normal copy of the affected gene ... Read more


SGT-001 is an experimental gene therapy being developed by Solid Biosciences to treat Duchenne muscular dystrophy (DMD), a type of muscular dystrophy characterized by progressive muscle weakness and atrophy. ... Read more

SRP-9001 Micro-Dystrophin

SRP-9001 micro-dystrophin is a gene therapy candidate for the treatment of Duchene muscular dystrophy (DMD). Scientists from Nationwide Children’s Hospital developed SRP-9001 micro-dystrophin. The potential treatment is licensed to ... Read more