Jacqueline Glascock of Cure SMA Discusses FDA-Approved SMA Treatments & Dissects Combination Therapy

The higher dose led to a more rapid decline in an important marker of neurodegeneration, according to a Biogen news release announcing the results.

Study of 29 children treated with Zolgensma (onasemnogene abeparvovec) shows mixed results.

U.S. providers surveyed said that getting insurance approval is time-consuming and can delay treatment for newborns with spinal muscular atrophy.

Data from the DEVOTE study suggest that 50-milligram doses are safe and effective.

Apitegromab, an investigational drug for spinal muscular atrophy, demonstrated sustained patient benefits and maintained safety over a three-year period in a phase II study, with 88% of participants maintaining or improving motor milestones.