The Giving Season is Here!
Cure SMA leads the way to a world where everyone impacted by SMA is empowered to lead their best lives. As 2022 comes to a close, we celebrate stories of breakthroughs powered by ...
Community Spotlight: Michelle Tynski and Family
Play is important for kids. It allows children to develop cognitive, social, emotional, and physical skills while engaging creatively. But it’s not just how kids play that’s important, what they play with also ...
Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)
Genentech, announced earlier this month new two-year data from the JEWELFISH study evaluating Evrysdi® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 months to 60 years at time of enrollment. ...
SMA Community Seeks SSI Improvements on Program’s 50th Anniversary
Fifty years ago today, the Supplemental Security Income (SSI) program was created to “provide supplemental security income to individuals who have attained age 65 or are blind or disabled” for their basic living ...
New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA
Scholar Rock yesterday announced new quality-of-life (QoL) data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes after 24 months of treatment which indicate stabilization or continued improvement with apitegromab for ...
Community Spotlight: Amber-Joi Watkins and Family
The Igbo and Yoruba proverb, “It takes a village to raise a child” has been in use for centuries and remains in use across languages and cultures due to its continued relevance. For ...
Cure SMA Holds Patient-Led Listening Session with FDA
On Thursday, August 4, 2022, six members of the SMA community had the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) about their lived experiences with SMA and their ...
A Deliciously Easy Way to Fundraise for Cure SMA
Nothing brings people together more than sharing a meal, but what if you could make that meal even more meaningful? We’re encouraging all members of our SMA community to consider hosting a dining ...
Special NBS Awareness Month Announcement: South Carolina Screens for SMA!
Cure SMA is pleased to announce that South Carolina is now screening babies born in the state for spinal muscular atrophy (SMA). South Carolina Department of Health and Environmental Control (DHEC) officials shared ...
SMA Community Update from Novartis Gene Therapies
Novartis Gene Therapies has provided an update to the SMA Community. Check out the latest here!
Kids: We Invite You to Bake a Difference for Cure SMA!
Cure SMA is calling all future bakers and pastry chefs to help us bake a difference for individuals with spinal muscular atrophy (SMA). Baking a Difference was created by kids for kids with ...
Seat Elevators on Power Wheelchairs are a Necessity, not a Luxury, according to the SMA Community
Individuals with spinal muscular atrophy (SMA) who utilize power wheelchairs for their mobility agree that seat elevation, which allows an individual to adjust their wheelchair seat, helps improve mental and physical health, increase ...