Sarepta to hunt approval for gene remedy in uncommon muscular dystrophy
An experimental gene remedy from Sarepta Therapeutics elevated ranges of the gene lacking in an ultra-rare type of muscular dystrophy, in line with information the corporate offered Friday.
The corporate has mentioned it plans to file for approval within the illness, generally known as limb-girdle muscular dystrophy (LGMD) 2E. That might make it the primary authorised remedy in LGMD, a broad assortment of extremely uncommon illnesses that may deprive sufferers of the power to stroll and in some instances shorten life. However it’s prone to face a major uphill battle.
The LGMD 2E remedy depends on the identical gene-ferrying virus that Sarepta makes use of in its different remedies, together with its authorised gene remedy for Duchenne muscular dystrophy, Elevidys, and experimental gene therapies for a number of different LGMD subtypes.
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