STAT Plus: Sarepta’s gene therapy for limb-girdle disease shows improved muscle function after one year
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.
Continue to STAT Plus to read the full story…
