“With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of our BLA on behalf of patients and families living with SMA.” Today, Scholar Rock announced the submission of a Biologics License Application (BLA) to the […]
“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 »
