“We are committed to supporting individuals with SMA and their families by advancing research that aims to answer critical questions for the community.” Today, Biogen announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and the European Medicines Agency (EMA) has validated the application for a […]
Cure SMA warmly invites you to join us at one of our 2025 Walk-n-Roll events—where community meets impact! This year, for the first time, you can register and start fundraising for both spring and fall events right now. Walk-n-Roll isn’t just a fundraiser; it’s a celebration of progress, hope, and togetherness. It’s a chance to
All 2025 Walk-n-Roll Events Are Open Read More »
On January 4th, 2025, President Biden signed the Think Differently Database Act (H.R. 670) into law, marking a significant legislative victory for the SMA community. This law will create an interactive, searchable webpage providing federal, state, and local information about caregiving services, including Medicaid Home and Community-Based Services (HCBS) eligibility, to individuals with disabilities and
Cure SMA Caregiving Priority Signed into Law Read More »
Novartis today announced positive topline results from the Phase III STEER study. This pivotal study assessed the efficacy and safety of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged two to less than 18 years who are able to sit but have never walked independently. Efficacy and
As 2024 draws to a close, Cure SMA reflects on an extraordinary year of advocacy milestones that have advanced the priorities of individuals with spinal muscular atrophy (SMA) and their families. From policy wins to meaningful events, we celebrate the SMA community for their role in the following highlights: 100% of States Screening Newborns for
Cure SMA Advocacy Highlights of 2024 Read More »
Cure SMA is thrilled to share a major win for the SMA community: the U.S. Department of Transportation (DOT) has finalized its rule on ensuring safe accommodations for air travelers with disabilities using wheelchairs. This significant advancement underscores the importance of equity in air travel and represents a critical step toward making flights safer and
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In the RESILIENT SMA study, Biohaven announced today that taldefgrobep alpha showed clinically meaningful improvements in motor function at all time points on the Motor Function Measurement-32 scale (MFM-32), but the treatment arm did not statistically separate on the primary outcome at Week 48 compared to the placebo+standard of care (SOC) group. Biohaven plans to
Individuals with spinal muscular atrophy (SMA) and their families recognized congressional champions and celebrated advocacy progress at Cure SMA’s Hope on the Hill event. U.S. Representative Sam Graves (MO-6) Photo: Jay Mallin U.S. Representative Pete Stauber (MN-8) Photo: Jay Mallin U.S. Representative Mark DeSaulnier (CA-10) Photo: Jay Mallin This year’s event, which was held on
SMA Community Advocacy Wins and Champions Celebrated at Cure SMA Event Read More »
Thank you for being an essential part of this journey and for believing in our mission. As we mark 40 years of dedicated service to the spinal muscular atrophy (SMA) community, we reflect on our journey and the incredible milestones we’ve achieved together. In 1984, a small group of parents founded Families of Spinal Muscular
Celebrate 40 Years of Impact – Join Us in Making a Difference! Read More »
As part of our ongoing efforts to drive quality health care standardization and improvements for the SMA community, Cure SMA is pleased to announce the next publication in a series of resources designed to support healthcare professionals, and individuals with SMA, and their caregivers. “Spinal Muscular Atrophy (SMA) Update in Best Practice: Recommendations for Treatment
