One of Cure SMA’s top priorities is to relay the SMA community’s treatment experiences and preferences to the United States Food and Drug Administration (FDA). This helps the FDA make patient-centered decisions about new SMA drugs. This is a critical time for patient input on SMA treatment, as a variety of new “add-on” therapies and […]
“Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval marks another significant step forward,” said Levi Garraway, M.D., Ph.D., Genentech’s Chief Medical Officer and Head of Global Product Development. “The Evrysdi tablet combines established efficacy with convenience, providing an
To address the chronic health needs of individuals with spinal muscular atrophy (SMA), Cure SMA and the SMA community have launched a new advocacy campaign in support of federal research for SMA. Through the campaign, Cure SMA seeks dedicated SMA research funding through a federal research program managed by the U.S. Department of Defense (DOD).
Cure SMA Launches Advocacy Campaign to Secure New Federal Research for SMA Read More »
February marks Rare Disease Month, a time to amplify the voices of the 300 million people worldwide living with a rare disease—including the individuals in the U.S. impacted by spinal muscular atrophy (SMA). Throughout this month, and especially during Rare Disease Week (starting February 23rd), we’re committed to advancing research, raising awareness, and advocating for
Rare Disease Month 2025: Driving Progress Through Research Read More »
“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 »
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 »
