Access to timely treatment for spinal muscular atrophy (SMA) remains a challenge, with barriers including delays in diagnosis, frequent treatment needs, and insurance coverage gaps, which impact ...
Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...
FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration challenges, with key selection factors like patient age and gene copy ...
Imagine a child born in a family with a rare disease, whose treatment would cost more than 17 crore for a single dose. The ...
After up to 4.8 years of continuous treatment with SPINRAZA, 100 percent of children treated pre-symptomatically were alive, and none require permanent ventilation Patients continued to maintain and ...
The first program I want to go over is the use of taldefgrobep alfa, which is being developed in the phase 3 RESILIENT study for the treatment of patients with spinal muscular atrophy. Before going ...
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile ...
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 ...
Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...
Infants up to 6 weeks old with genetically diagnosed presymptomatic spinal muscular atrophy (SMA) treated with oral risdiplam (Evrysdi) appeared to have better functional and survival outcomes ...
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