McGill researchers discovered that K884, an experimental drug, enhances muscle stem cell repair in Duchenne muscular dystrophy (DMD). Unlike gene therapies, K884 targets DMD-affected cells regardless ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...

A gene therapy candidate extended survival in a mouse model of SMARD1, supporting its testing in a clinical trial for this ...

A new gene therapy for Duchenne muscular dystrophy (DMD) has shown promise in not only slowing the progression of the disease but potentially even reversing the muscle damage, with human trials set to ...

(MENAFN- GetNews) (Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Spinal Muscular Atrophy pipeline constitutes 18+ key companies continuously working towards developing ...

(MENAFN- GetNews) (Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Spinal Muscular Atrophy pipeline constitutes 18+ key companies continuously working towards developing ...

Gene therapy approach developed offers new hope for those with Duchenne muscular dystrophy by restoring the full-length dystrophin protein. Systemic delivery of full-length dystrophin in Duchenne ...

Spinal Muscular Atrophy companies are Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst ...

SMA is the leading genetic cause of infant mortality. However, due to its extremely low incidence rate, the disease becomes a low priority for most pharma manufacturers due to the diminished market ...