Watch the video and see how far we’ve come and the future we’re building together. Donate to Support the SMA Community Your support accelerates research, strengthens advocacy, and expands critical family services for individuals and families living with SMA. Make your impact today.
Together, We’re Changing the Story of SMA Read More »
Earlier this year, Cure SMA launched an expanded Phase 10 of the SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, SMA Europe, Cure SMA, and other nonprofit organizations to share information, ideas, and data to benefit the broader SMA community. Through the Industry Collaboration, we fund projects to ensure
Cure SMA Shares Updates on the Phase 10 SMA Industry Collaboration Read More »
Today is Giving Tuesday, and there’s an incredible opportunity to double your impact! The Luke 18:1 Foundation has stepped forward with a $50,000 gift and invited our community to double the impact in the next 24 hours. By giving today, you help us rise to this special Giving Tuesday challenge and bring meaningful support to children,
Double Your Impact this Giving Tuesday! Read More »
Novartis today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Itvisma (onasemnogene abeparvovec-brve) to treat adults and children 2 years of age and older with spinal muscular atrophy (SMA). Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that works by delivering a functional copy of the SMN1 gene directly to motor neurons, helping the body restore
Novartis Receives FDA Approval of Itvisma for the Treatment of SMA Read More »
Read the Scholar Rock community statement here. Scholar Rock recently held an in-person Type A meeting with the FDA on November 12 to discuss the Biologics License Application (BLA) for apitegromab, a potential treatment for children and adults with SMA. Cure SMA was present and active at this FDA meeting to represent our
Scholar Rock and Cure SMA Share Update on Apitegromab and FDA Meeting Read More »
Novartis recently released an update on their continued collaboration with the SMA community through fundraising events and community support programs, and they will continue to provide timely updates as they become available. Read the full community update letter here. “Over the past few months, we’ve been inspired by the continued strength, unity, and advocacy across
Novartis Releases Fall SMA Community Update Letter Read More »
As 2025 starts to wind down, Cure SMA is proud to launch our year-end fundraising campaign: Unlock a Future of Possibilities. This theme represents hope, progress, and the life-changing difference your support makes for individuals and families impacted by spinal muscular atrophy (SMA). At the heart of this campaign are four members of the SMA community
Unlock a Future of Possibilities: Cure SMA Launches Year-End Campaign Read More »
Yesterday, Scholar Rock shared an SMA community update letter on their Biologics License Application (BLA) for apitegromab in SMA. As shared on September 23, 2025, Scholar Rock received a Complete Response Letter (CRL) from the FDA for the apitegromab BLA in SMA. As a reminder, the CRL was solely related to manufacturing observations identified at
Scholar Rock Releases SMA Community Update Letter on Apitegromab Read More »
Today, Biogen announced plans to initiate Phase 3 development to evaluate the effectiveness and safety of salanersen, an investigational drug for the treatment of spinal muscular atrophy (SMA). Phase 3 initiation follows the announcement of positive interim results of the Phase 1 study of salanersen, which informed the design of the studies. Biogen is preparing
Biogen to Initiate Phase 3 Pivotal Study Evaluating Investigational Drug Salanersen Read More »
Cure SMA has updated its Health Insurance Road Map to help patients, families, and providers navigate the complexities of health insurance. The updated resource reflects the current healthcare landscape and offers essential tools to simplify challenging insurance topics and promote informed decision-making. The Health Insurance Road Map includes: Clear definitions of commonly used insurance terms
Cure SMA Updates Health Insurance Road Map to Support Patients and Families Read More »
