STAT+: Sarepta to seek approval for gene therapy in rare form of muscular dystrophy

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason on Signal at JasonMast.05.

An experimental gene therapy from Sarepta Therapeutics increased levels of the gene missing in an ultra-rare form of muscular dystrophy, according to data the company presented Friday.

The company has said it plans to file for approval in the disease, known as limb-girdle muscular dystrophy (LGMD) 2E. That would make it the first approved treatment in LGMD, a broad collection of highly rare diseases that can deprive patients of the ability to walk and in some cases shorten life. But it is likely to face a significant uphill battle. 

The LGMD 2E therapy relies on the same gene-ferrying virus that Sarepta uses in its other treatments, including its approved gene therapy for Duchenne muscular dystrophy, Elevidys, and experimental gene therapies for several other LGMD subtypes.