Scientists have been searching for ways to cure DMD for as long as they have known about the disease. Today, based on the wealth of knowledge about the underlying genetic and molecular mechanisms ...

SGT-003 gene therapy showed significant microdystrophin expression and muscle integrity improvements in DMD patients, with no serious adverse events reported. The novel AAV vector demonstrated ...

An experimental gene therapy for Duchenne Muscular Dystrophy (DMD) has showed better-than-expected results in a three-patient trial, according to preliminary data. Company shares jumped 60 percent ...

Solid Biosciences Inc. is preparing for a sit-down with the U.S. FDA this year to discuss the firm’s results with the next-generation gene therapy SGT-003 for Duchenne muscular dystrophy (DMD).

Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused by mutations in the DMD gene. Here, the authors develop MyoAAV-UA, a compact utrophin activator, as a promising universal ...

Solid Biosciences’ gene therapy candidate for patients with Duchenne muscular dystrophy (DMD) has elicited a 110% expression of the microdystrophin gene. The US-based company’s stock soared by ...

Duchenne muscular dystrophy is a genetic disorder caused by mutations in the DMD gene, resulting in a lack of the protein dystrophin. This deficiency leads to progressive muscle weakness and loss ...