Genentech recently issued an SMA community statement highlighting updates from the 2023 SMA Research & Clinical Care Meeting. These updates included: Investigating risdiplam treatment post gene-therapy Long-term efficacy and safety of risdiplam Real-world evidence generation Check out the full community statement here.
Genentech Releases SMA Community Letter Read More »
On July 27th, the U.S. Senate Appropriations Committee supported a Cure SMA-requested provision asking the National Institutes of Health (NIH) to address the unmet needs of individuals with spinal muscular atrophy (SMA) through new SMA research. The provision, which can be found on pages 132 & 133 of the fiscal year 2024 Labor-Health and Human
Key Congressional Committee Urges NIH to Address Unmet Needs of Individuals with SMA Read More »
Whether you have SMA, have a loved one with SMA, are a researcher, care provider, industry partner, or are new to the SMA community, SMA Awareness Month is a time to come together to celebrate, honor, reflect, remember, and learn. The theme of SMA Awareness Month 2023 is ‘Breakthroughs Begin with You.’ It’s an opportunity
August is SMA Awareness Month! Read More »
Cure SMA is pleased to announce the launch of an expanded Phase 3 of the Real World Evidence Collaboration. The collaboration was established to leverage the experience, expertise and resources of pharmaceutical and biotechnology companies and nonprofit organizations involved in development of SMA therapeutics to guide the future direction of real world evidence collection and
Cure SMA Announces Expanded Phase 3 of Real World Evidence Collaboration Read More »
On the anniversary of the Americans with Disabilities Act, the U.S. Department of Transportation (DOT) announced a final rule that will require airlines to take short- and long-term action to make lavatories on new single-aisle aircraft more accessible for people with disabilities, including those who fly in a wheelchair. “The inability to safely access and
New DOT Rule Will Require Truly Accessible Lavatories on New Single Aisle Aircraft Read More »
At the end of June, SMA researchers and clinicians from around the world met in Orlando, Florida, for the 2023 Annual SMA Research & Clinical Care Meeting. The annual meeting provides an opportunity for SMA scientists and clinicians to share their most recent discoveries and learnings, and is an incubator for collaborations, including cross-disciplinary
Thank you to everyone who attended the 2023 Annual SMA Conference at Disney’s Yacht & Beach Club in Orlando, Florida! It was an impactful weekend of opportunities to connect, learn, and have fun. We are happy to announce that, including on-site registration, there were over 3,200 attendees in total – a record for the Annual
Thank You for an Amazing 2023 Annual SMA Conference! Read More »
This week, bills to address everyday challenges faced by individuals with spinal muscular atrophy (SMA) took important steps forward in the U.S. Congress. A key aviation bill that includes a section that will make air travel safer and more accessible for passengers with disabilities, including SMA, was approved by the U.S. House of Representatives by
Big Week in Washington for Cure SMA-Supported Legislation Read More »
Nieves and Valerie Gomez’s SMA story started in 2013, when their son Liam Taylor Gomez was born with SMA Type 1. Liam passed away at six months of age, and at that time Nieves made a commitment to dedicate the rest of his life to healthcare and helping others. Since then, he has worked in
Donor Spotlight: Nieves and Valerie Gomez Read More »
Cure SMA is pleased to announce the launch of the second annual State of SMA report. The purpose of this report is to share highlights from Cure SMA’s three databases: a patient-reported database with data from over 9,700 affected individuals worldwide that also incorporates longitudinal data from our annual community update survey; the SMA clinical
Cure SMA’s 2022 State of SMA Report Read More »
