Sarepta Therapeutics (NASDAQ:SRPT) perks up 2% premarket on light volume on the heels of updated follow-up data on two of its gene therapy candidates.
SRP-9003 in limb-girdle muscular dystrophy: 18-month functional data from three subjects in the low-dose cohort and six-month functional data from three participants in the high-dose arm showed improvements from baseline across all functional measures. No new treatment-related safety signals have been observed.
SRP-9001 in Duchenne muscular dystrophy (DMD): Two-year follow-up results from four patients showed a mean increase of 7.0 points (5.5 points at year one) from baseline in a scale measuring functional motor abilities in ambulatory DMD patients called NSAA (scale range is 0-34).
Development is ongoing.
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