Yesterday WSJ published an article about new gene therapy developed by Sarepta Therapeutics -Golodirsen (SRP-4053) – to treat Duchenne Muscular Dystrophy (DMD). DMD is a progressive X-linked recessive neuromuscular disorder which is characterized by a progressive degeneration of skeletal, smooth and cardiac muscles, and which results in diffuse muscle weakness. Patients with DMD rarely live beyond 25 years. The disease worsens due to a lack of the dystrophin protein in skeletal muscles, along with the increased blood level of an enzyme leading to muscle tissue degeneration.
Sarepta has already conducted a therapy clinical trial on 3 patients with very positive results. Positively, Golodirsen triggers a 38% increase in dystrophin levels. Despite a situation of restricted statistical information, WSJ article writer hopes FDA will bear the risk and support the therapy approval. In such a case, Sarepta’s new therapy could become the first effective treatment available to young patients diagnosed with DMD. If so, the company’s revenues are likely to grow, thus allowing further research on this rare disease.