"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...
Please provide your email address to receive an email when new articles are posted on . An investigational, fully human monoclonal antibody developed as a muscle-targeted therapy for spinal muscular ...
A Prescription Drug User Fee Act target date of September 22, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...
The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.
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 ...
Please provide your email address to receive an email when new articles are posted on . At 5 years, 91% of pediatric patients treated with Evrysdi were alive, 81% without permanent ventilation. Data ...
The high-dose regimen of SPINRAZA (nusinersen) is approved in the European Union and Japan. The high-dose regimen is under review with the United States Food and Drug Administration (FDA) and has a ...
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 ...
Biogen said that new data examining its already approved spinal muscular atrophy treatment Spinraza (nusinersen) indicates that a higher dose than currently approved may lead to better improvement in ...
Scholar Rock’s stock soars more than 315% on positive trial of treatment for spinal muscular atrophy
Scholar Rock Holding Corp.’s stock soared more than 315% Monday to put it on track for its biggest ever one-day gain, after the clinical-stage biotech company said a late-stage trial of a treatment ...
On Wednesday, Novartis AG (NYSE:NVS) announced safety and efficacy results from the Phase 3 program for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad population of ...
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