Smanewstoday.com
AAN 2023: Spinraza shows benefits in clinical trial of SMA adults
WebTo learn more about how treatment with Spinraza affects adults with milder forms of the disease, the Washington University School of Medicine, in St. Louis, Missouri, is sponsoring a clinical trial called Spinraza in Adult SMA, or SAS (NCT03709784). “The SAS trial … is designed to look at the safety and efficacy of [Spinraza] over 30 months in …
Actived: 9 days ago
URL: smanewstoday.com
Zolgensma for SMA SMA News Today
(5 days ago) WebA one-time gene therapy, Zolgensma (onasemnogene abeparvovec-xioi) is widely approved for the treatment of spinal muscular atrophy (SMA). It was the second SMA treatment ever to be approved in the U.S. and the first approved gene therapy for the rare disease. Zolgensma was originally developed by AveXis, now known as Novartis Gene …
SMA News Today Home SMA News Today
(5 days ago) WebThe Muscular Dystrophy Association (MDA) has opened the MDA Gene Therapy Support Network — a new program it’s dubbed GTx — to aid and educate families living with spinal muscular atrophy (SMA) or other neuromuscular diseases (NMDs) who may be considering a gene therapy as part of…. May 25, 2023 Columns by Ari Anderson.
SMA Life Expectancy and Disease Onset SMA News Today
(5 days ago) WebSMA Life Expectancy and Disease Onset. Spinal muscular atrophy (SMA) is a progressive genetic disorder that affects the nervous system and muscles, and is a very rare disease at that, found in an estimated 1 in every 6,000 to 1 in every 10,000 people. It is caused by a loss of specialized nerve cells, called lower motor neurons, leading to
SMA Gene Therapy SMA News Today
(2 days ago) WebZolgensma (onasemnogene abeparvovec-xioi) is the only gene therapy currently approved to treat SMA. It is marketed by Novartis, and administered via a single infusion into the bloodstream. The therapy uses a harmless, lab-engineered version of adeno-associated virus serotype 9 (AAV9) to deliver its genetic payload to the body’s cells.
Apitegromab (SRK-015) for Spinal Muscular Atrophy (SMA)
(5 days ago) WebApitegromab (SRK-015) is an experimental muscle-directed therapy being developed by Scholar Rock for spinal muscular atrophy (SMA). It is designed to improve motor abilities in SMA patients. As a muscle-directed therapy, apitegromab’s mechanism of action is distinct from currently approved disease-modifying therapies for SMA, all of …
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