Sarepta Therapeutics (NasdaqGS:SRPT) reported positive three year functional results from its Phase 3 EMBARK trial of Elevidys in ambulatory Duchenne muscular dystrophy patients. The data show slowed ...

Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...

Scientists have created a new gene therapy for Duchenne muscular dystrophy (DMD) that may not only help stop the disease in DMD patients, but might also help restore their damaged muscles in the ...

Sarepta stock jumped Monday after the company unveiled three-year test results for its embattled gene therapy, Elevidys.

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the ...

The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the risks and benefits of the drug The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the ...

Sarepta says effects of Duchenne muscular dystrophy gene therapy were durable three years after treatment; More biotech ...

A mother's love drives her to pursue experimental treatment as two of her four children battle a rare genetic disease that ...

The U.S. Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...