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 ...
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 ...
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 ...
The Chosun Ilbo on MSN
Gene therapy miracle: British boy walks, swims
A 5-year-old British boy with 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 BBC ...
Please provide your email address to receive an email when new articles are posted on . The study included 58 individuals with SMA types 2 and 3 who were treated with IV apitegromab for 52 weeks. At ...
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 ...
(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) -- ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback