Sarepta Therapeutics said Monday that three children born with a rare muscle-wasting disease called limb-girdle muscular dystrophy are showing durable improvements in strength and function a year after a one-time infusion of an experimental gene therapy.
Three more children have been treated with a higher dose of the same gene therapy. Initial biopsy results from those patients, also reported Monday, show greater production of a key muscle protein with no added safety problems — suggesting the higher dose could lead to even larger improvements in muscle function. Those tests will be performed later this year, Sarepta said.
Sarepta’s next step is to reach an agreement with the U.S. Food and Drug Administration on the design and scope of a pivotal clinical trial that could start early next year.
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I am lgmd 2b patient plz helf mi