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 ...
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 ...
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, 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 ...
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British boy walks, swims after gene therapy
A 5-year-old British boy suffering from the rare disease spinal muscular atrophy (SMA) has miraculously started walking four years after receiving the world’s most expensive gene therapy. On the 31st, ...
The cost burden of patients with SMA is considerable, and is estimated to be approximately $4 million to $5 million over 10 years in patients with early-onset SMA. This cost is 54.2 times greater than ...
(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) -- ...
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 ...
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