Spinal muscular atrophy is a genetically inherited disorder that causes muscle weakness. Adults can get spinal muscular atrophy, but it’s rare in adults and progresses slowly. It doesn’t typically ...
- Motor function improvements were observed for all three SRK-015 treatment cohorts in the primary efficacy endpoints (Hammersmith scale scores) at six-months; top-line data from the 12-month ...
Some patients with later-onset spinal muscular atrophy (SMA) type 2 and type 3 had improved motor function when the investigational monoclonal antibody apitegromab was added to their treatment, the ...
Evrysdi is a survival motor neuron-2 (SMN2) splicing modifier. The Food and Drug Administration (FDA) has approved Evrysdi™ (risdiplam; Genentech) for the treatment of spinal muscular atrophy (SMA) in ...
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented 1-year data from the pivotal Part 2 of SUNFISH, a global ...
Positive CHMP opinion is based on data from the DEVOTE study which evaluated the high dose regimen of nusinersen in treatment-naive participants ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Back to Healio Topline results from a phase 3 clinical ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Patients treated with onasemnogene abeparvovec recorded ...
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