Cure SMA, a nonprofit organisation supporting those impacted by spinal muscular atrophy (SMA), on Monday welcomed the announcement by biopharmaceutical company Scholar Rock (NASDAQ:SRRK) of FDA approval of ISEMBYLD (apitegromab-mstn) to treat people living with SMA.

This approval for treatment of adults and children aged 2 years and older brings the first muscle-targeted treatment to the SMA community.

ISEMBYLD 10 mg/kg is administered once every 4 weeks as an intravenous (IV) infusion by a healthcare professional at an infusion centre, hospital, or at home.

Cure SMA will host a community webinar on 30 September 2026 to provide education and guidance on ISEMBYLD, including next steps for ensuring broad access, and practical resources to support individuals and families in making informed decisions with their healthcare teams.