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Sarepta Therapeutics’ DMD gene therapy shows early success

May 19, 2021


The 12-week results come from the first 11 participants enrolled in Sarepta’s Study SRP-9001-103 or ENDEAVOR study, which is being conducted in partnership with Swiss pharma company Roche.

The study, which has so far enrolled 20 participants with DMD between the ages of four to seven, is aiming to determine the change from baseline in the quantity of micro-dystrophin protein expression at 12 weeks following administration with the gene therapy, SRP-9001.

SRP-9001 is an investigational gene therapy designed to deliver the micro-dystrophin-encoding gene to muscle tissue to boost production of the micro-dystrophin protein.

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