Recent clinical trial results for SRP-9001 show high levels of sustained improvements in patients suffering from Duchene muscular dystrophy (DMD). This debilitating disease is caused by a mutation in the dystrophin gene, a protein vital to the function of muscles. Patients suffer progressive muscle weakness and degeneration and are typically wheelchair bound as children.
Clinical trials of Serepta Therapeutic's gene therapy candidate, SRP-9001 have all shown sustained improvements in motor function due to delivery of micro dystrophin, a version of the dystrophin gene, using a recombinant AAV under the control of MHCK7, a muscle specific promoter.
This provides positive news for patients suffering from this otherwise untreatable disease, worldwide.
SRP-9001 Micro-Dystrophin - Muscular Dystrophy News
Sustained Gains for Duchenne Boys Evident in Gene Therapy Trials (musculardystrophynews.com)