CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...

Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...

The MDA's 75th anniversary conference highlights advancements in gene therapy, precision medicine, and patient-centered care for muscular dystrophy. Discussions focus on equitable access to ...

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of individuals with Types 2 or 3 spinal muscular atrophy (SMA) Late-breaking ...

Reflex sympathetic dystrophy is a painful, disabling disorder of unknown pathophysiological origin that usually commences after trauma to or surgery on a limb. In chronic cases, the syndrome leads to ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Patients treated with onasemnogene abeparvovec recorded ...

Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...

Discusses 3-Year Topline Results From EMBARK Phase 3 Trial of ELEVIDYS in Duchenne Muscular Dystrophy January 26, ...

Oral presentation to include additional efficacy data from the Phase 3 SAPPHIRE trial of apitegromab in patients with spinal muscular atrophy (SMA) Poster presentations include preclinical data on a ...