Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

The MarketWatch News Department was not involved in the creation of this content. New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and ...

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

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 York, Feb. 12, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today celebrates the decision by the US Food and Drug Administration (FDA) to grant approval of a risdiplam ...

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 ...

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 ...

Biogen offers a financial assistance program for its treatment Spinraza but there’s a catch: patients must sign away their protected health information to the company, reports the Waco Tribune-Herald.

Jesy Nelson reveals her twins may never walk, but what is spinal muscular atrophy? - Jesy Nelson reveals her twin daughters have been diagnosed with SMA, a severe muscular disorder ...

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 ...

New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed ...

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 ...