Front Page News
Scholar Rock Receives Approval for ISEMBYLD™ for the Treatment of SMA
(opens in new tab) Scholar Rock today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for ISEMBYLD™ (apitegromab-mstn) to […]
Genentech Shares Highlights from an Unforgettable Week at the Annual SMA Conference
(opens in new tab) From a lively “Evryday Café” exhibit hall experience to an advocacy reception featuring conversations on policy and access, Genentech recaps a […]
Novartis Highlights Accessible Travel Partnership in Summer Community Update
From their time at the Cure SMA Annual Conference, to their SMAshing My Limits partnership with Becoming rentABLE, opening up accessible beach houses, treehouses, and […]
Newly published research from Cure SMA: What helps young adults with SMA successfully transition to adult care?
As increased treatment options have helped more people with spinal muscular atrophy (SMA) live longer, healthier lives, more young adults are navigating the transition to […]
Thank you to everyone who attended the 2026 Annual SMA Conference
Thank You Thank you to everyone who attended the 2026 Annual SMA Conference at Disney’s Yacht & Beach Club Hotel in Orlando, Florida! It […]
Biogen Shares Conference Highlights and New High Dose SPINRAZA Resources
From the High Dose SPINRAZA symposium to a family-friendly poster session, Biogen recaps their experience at this year’s Annual SMA Conference and highlights new tools […]
Visit the Cure SMA Hub at This Year’s Annual SMA Conference
The Cure SMA Hub is your go-to space at the Annual SMA Conference to learn about key Cure SMA programs and opportunities for involvement. Stop […]
FDA Grants Breakthrough Therapy Designation to Biogen’s Salanersen for Spinal Muscular Atrophy
Biogen recently announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to salanersen, an investigational therapy being studied for the […]
NMD Pharma Releases Community Letter Regarding Phase 2 SYNAPSE-SMA Clinical Trial
NMD Pharma recently shared exciting news with the SMA community: the last participant has completed their part in the SYNAPSE-SMA Phase 2 clinical trial, testing […]
Meet the Scientists Behind SMA Research and Discover What’s Coming Next
The Family-Friendly Poster Session is one of the most anticipated events at the Annual SMA Conference! This year’s session will be held on Friday, June […]