One of Cure SMA’s top priorities is to relay the SMA community’s experiences, preferences, and unmet needs to the United States Food and Drug Administration (FDA). This enables the FDA to make patient-centered decisions about new SMA drugs. This is a critical time for patient input on SMA treatment, as new therapies and variations […]
Cure SMA Shares Patient Voice and Needs with FDA Read More »
Cure SMA completed a year-long advocacy effort to educate public and private insurers about the current state of spinal muscular atrophy (SMA), including the unmet needs of individuals with SMA and barriers they face in accessing essential healthcare and treatments. Patient and clinical-reported data have chronicled how SMA is rapidly changing due to the
Cure SMA Educates Insurers on Unmet Needs and Access Barriers of the SMA Community Read More »
A key congressional committee approved fiscal year 2026 legislation that now prioritizes new federal research funding for spinal muscular atrophy (SMA). The U.S. Senate Appropriations Committee recognized the unmet medical needs of individuals with SMA, including those from military and veteran families, by this year adding “spinal muscular atrophy” within a biomedical research program managed
Key Congressional Committee Recommends New Federal Research in SMA Read More »
Whether you live with SMA, know someone who does, work in research or care, or are beginning your journey with the community—SMA Awareness Month is a time to come together to celebrate progress, honor experiences, reflect on challenges, and build momentum for the future. The theme of SMA awareness month, “Breakthroughs Begin with You,” puts
This August is Spinal Muscular Atrophy (SMA) Awareness Month! Read More »
Thank you to everyone who attended the 2025 Annual SMA Conference at the Disneyland Hotel in Anaheim, California! It was an impactful weekend of opportunities to connect, learn, and have fun. We’re thrilled to share that nearly 2,800 attendees joined us – our second-largest conference to date! It was an honor to have individuals with
Thank You for an Amazing 2025 Annual SMA Conference! Read More »
At this year’s Annual Cure SMA Conference in Anaheim, California, more than 2,800 attendees from across the country came together to learn, connect, and imagine a better future—including a future where air travel is safe, accessible, and dignified for all wheelchair users. While progress is being made, many individuals and families in the SMA community
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.
Cure SMA Awards $140,000 Grant to Mandana Arbab, PhD, at Boston Children’s Hospital Read More »
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.
“To see a child dosed with gene therapy at age one and still unable to sit at age five then gain that ability within three months of starting salanersen is unexpected and encouraging,” – Dr. Valeria Sansone, principal investigator of the Phase 1 trial Biogen Inc. announced today that it will advance its investigational
