STAT+: Sarepta to seek approval for gene therapy in rare form of muscular dystrophy
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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.<\/p>\n
The company has said it plans to file for approval in the disease, known as limb-girdle muscular dystrophy (LGMD) 2E.\u00a0That 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.\u00a0<\/p>\n
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<\/a>, and experimental gene therapies for several other LGMD subtypes.\u00a0<\/p>\n Continue to STAT+ to read the full story\u2026<\/a><\/p>\n<\/div>\n