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Sarepta shares rocket 55% on positive early results for DMD gene therapy
JAY GORY, MANAGING EDITOR, science
Sarepta Therapeutics Inc. shares rocketed 55.6% in extremely heavy Tuesday morning trade after the company reported positive early results from a phase 1/2a gene therapy clinical trial for three patients with the rare disease Duchenne muscular dystrophy. The three patients, the first ones enrolled in the study, showed "robust" expression of micro-dystrophin, a shortened version of the dystrophin gene used in gene therapy, Sarepta said. Individuals with DMD have a mutation on their dystrophin gene and thus cannot produce the protein dystrophin, which is critical for muscular development. read more


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