SMA Community Research and Legislative Priorities Finalized in New Federal Law

On February 3rd, Congress approved and the President signed into law key research and legislative priorities that Cure SMA and individuals with spinal muscular atrophy (SMA) and their families have been advocating for over the past year. The Consolidated Appropriations Act of 2026 includes the following provisions that will benefit children and adults with SMA […]

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Genentech Releases Winter SMA Community Update Letter

Genentech recently released an update on their continued collaboration with the SMA community and Evrysdi® tablet updates. They will continue to provide timely updates as they become available. Read the full community update letter here. “Looking back, 2025 was a time that was undeniably defined as a year of great change. We successfully navigated shifts,

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All 2026 Walk-n-Roll Events Are Open

Cure SMA warmly invites you to join us at one of our 2026 Walk-n-Roll events—where community meets impact! This year, 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 gather with others who

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Cure SMA Shares Updates on the Phase 10 SMA Industry Collaboration 

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

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Double Your Impact this Giving Tuesday!

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,

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Novartis Receives FDA Approval of Itvisma for the Treatment of SMA

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

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Scholar Rock and Cure SMA Share Update on Apitegromab and FDA Meeting 

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

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Novartis Releases Fall SMA Community Update Letter

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

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