Approval is supported by data from the DEVOTE study which showed the benefit of the SPINRAZA 50 mg and 28 mg regimen in both treatment-naïve and ...

Results from a single-center, observational, prospective study suggested that inflammatory markers could predict disease severity and treatment efficacy for patients with spinal muscular atrophy (SMA) ...

A retrospective, exploratory analysis identifies biomarkers predictive of spinal muscular atrophy (SMA) response to nusinersen and suggests the efficacy of using machine learning algorithms to ...

New data from Biogen shows improved motor function in SMA patients using a higher dose of nusinersen, highlighting early treatment benefits. Biogen announced new data from the DEVOTE Part C and ...

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

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...

New analyses from DEVOTE Part C further characterize the improvements in motor function in participants with SMA who transitioned to the investigational higher dose regimen of nusinersen from 12 mg ...

Spinraza is a medication used in children (including newborns) and adults to treat spinal muscular atrophy. Treatment begins with four loading doses, followed by one maintenance dose every 4 months.