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 for the community. Read their latest community update here.
Biogen Shares Conference Highlights and New High Dose SPINRAZA Resources Read More »
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 by to sign up for fall events, learn about advocacy, and connect with different Cure SMA teams. This year, we are also excited to feature special guest community volunteers who
Visit the Cure SMA Hub at This Year’s Annual SMA Conference Read More »
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 treatment of spinal muscular atrophy (SMA). This designation is intended to help speed the development and review of drugs that show early promise in treating serious conditions. Salanersen is an
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 ignaseclant (formerly known as NMD670), an investigational therapy designed to help strengthen skeletal muscles. The trial included 52 adults with SMA Type 3 at sites across the US and Europe.
NMD Pharma Releases Community Letter Regarding Phase 2 SYNAPSE-SMA Clinical Trial Read More »
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 26, from 6:30 pm to 8:30 pm in the Grand Harbor Ballroom at Disney’s Yacht and Beach Club Resort. The Family Friendly Poster Session is an opportunity for families to
Meet the Scientists Behind SMA Research and Discover What’s Coming Next Read More »
As more SMA treatments become available, people living with SMA, their families, and caregivers are asking new questions about how treatments work, whether using more than one treatment could help, and what to consider when thinking about combination therapy. Understanding how SMA treatments work can help individuals and families feel more informed about their options
Cure SMA is pleased to announce the participation of our Research Department staff in the following conferences: 2026 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference (March 8 – 11, 2026; Orlando, FL) 5th International Scientific Congress on SMA (March 11 – 14, 2026; Budapest, Hungary) 2026 American Academy of Neurology (AAN) Annual Meeting
Cure SMA is pleased to announce the publication of the 5th annual State of SMA report. Drawing on data collected by Cure SMA through 2025, this report features insights into the progress and ongoing challenges impacting people with spinal muscular atrophy (SMA) and their families. Previously, the annual report has also been used to advocate
Cure SMA Releases the 2025 Annual State of SMA Report Read More »
Today, Scholar Rock shared that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational treatment for spinal muscular atrophy (SMA), with a Prescription Drug User Fee Act (PDUFA) action date of September 30, 2026. This is a meaningful step forward in the path toward a potential new
Cure SMA has released a new policy brief highlighting the essential role Medicaid-funded home care plays in the lives of individuals with spinal muscular atrophy (SMA), a rare neuromuscular disease. Due to severe muscle weakness, many individuals with SMA rely on Medicaid-funded home care to meet their complex daily needs, including eating, bathing, bathroom support,
