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 ...

Muscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community The Muscular Dystrophy Association ...

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 ...

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 ...

(MENAFN- GlobeNewsWire - Nasdaq) The only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA). New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- ...

STATEN ISLAND, N.Y. -- Three-year-old Aubrey Paige Ibrahim is a beautiful little girl fighting a battle most adults would find challenging, if not impossible. Born on May 31, 2013, Aubrey was ...

At her eight-month pregnancy check-up, Lizmara Figueroa met with a new doctor. As he walked out the door, he made a parting comment. “He says, ‘Oh, just so you know, you’re a carrier for (spinal ...

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