Positive CHMP opinion is based on data from the DEVOTE study which evaluated the high dose regimen of nusinersen in treatment-naive participants ...

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 ...

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 ...

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 ...

- 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 ...

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 ...

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 ...

—The slow progression of SMA in adults and older children complicates evaluation of treatment response to newer therapies. Longitudinal quantitative MRI (qMRI) may give clinicians a better handle on ...

Spinal muscular atrophy (SMA) is an inherited disease that attacks motor neurons, the nerve cells that control our muscles. SMA hits children and adults. Children inherit the gene for SMA from both ...