BioCentury
ARTICLE | Clinical News

Limb-girdle muscular dystrophy gene therapy data lift Sarepta

October 4, 2019 8:48 PM UTC

Early data from a Phase I/II study of SRP-9003 to treat a form of limb-girdle muscular dystrophy showed the gene therapy led to functional improvements in all three patients in a low-dose cohort at nine months. Results from a planned higher-dose cohort would inform the design of a pivotal study; Sarepta intends to give guidance next year on a timeline.

Sarepta Therapeutics Inc. (NASDAQ:SRPT) shares climbed $6.91 to $88.24 on Friday, adding more than $500 million in market cap...

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