The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. A 51-year-old man died last month after ...

Researchers at the Experimental and Clinical Research Center (ECRC), a joint institution of the Max Delbrück Center and Charité—Universitätsmedizin Berlin, have developed a promising gene-editing ...

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 ...

Researchers at the USF Health Morsani College of Medicine have identified a potential pathway that could protect cardiac ...

SK pharmteco today announced details of its ongoing strategic manufacturing partnership with Genethon, a France-based pioneering nonprofit research and development organization dedicated to gene ...

The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...

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 ...

Most parents wouldn’t be thrilled with the idea of their kids getting hooked up to an IV bag filled with trillions of viruses. But for Melanie Hennick, whose son, Connor, has Duchenne muscular ...

Sonothera is developing a new way to deliver genes into cells that, if borne out, could have a big impact on muscular ...

When Chase Finazzo was just a few years old, his parents noticed Chase was pretty clumsy. But they didn't think it was anything serious. "He would fall a lot. Not like a lot a lot. But he had trouble ...

Liam Bendicksen is a research assistant, Aaron Kesselheim is the director and Edward Cliff is a research affiliate at the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital.