SMA Community Update from Novartis Gene Therapies
Novartis Gene Therapies has provided an update to the SMA Community. Check out the latest here!
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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 sweet hearts and a love of sweet treats. From a red wagon selling treats outside a local grocery store to full-fledged enterprise
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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 quality of life, and promote independence. The SMA community’s views were shared in a public comment letter Cure SMA sent to the
Cure SMA Launches Benefit-Risk Survey to Assess Patient Perspective on Treatment Possibilities and Risks
Cure SMA is conducting a survey to understand our community’s perspectives on treatment possibilities and tolerance for the side effects of currently available and planned therapies. The results will help inform our future objectives and priorities as well as help us continue to bring the patient voice to regulatory authorities. Some of you may recall
Cure SMA Care Center Network Site Expansion
The Cure SMA Care Center Network is excited to announce the addition of 9 NEW centers to the Care Center Network, bringing the current total number of participants in the Care Center Network to 27 sites across the US. The new centers of the Care Center Network: Baylor College of Medicine, Houston, Texas Children’s Healthcare
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Community Statement from Novartis Gene Therapies: Zolgensma Safety Monitoring Update on Acute Liver Failure
Dear SMA Community, Novartis Gene Therapies is committed to patient safety and the ongoing monitoring of adverse events as it relates to the use of Zolgensma® (onasemnogene abeparvovec-xioi), the one-time gene therapy for spinal muscular atrophy (SMA). We are writing to share that there have recently been two patient fatalities related to acute liver failure
Biogen Shares Community Statement for SMA Awareness Month
Learn about Biogen’s latest news in SMA by viewing their Community Statement (available as a PDF) and a related video.
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FDA Issues Safety Warning Regarding Neck Float Devices for Babies
On June 28,2022, the U.S. Food and Drug Administration (FDA) issued a safety communication about using neck float with babies. This occurs when a serious injury has been reported while using a device or product. The FDA warns parents, caregivers, and health care providers not to use neck floats with babies for water therapy interventions,
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Community Spotlight: Alexandra and Joe Lakhman
While sibling relationships aren’t always easy, Alexandra ‘Lexi’ Lakhman and her brother Joe have always been close. Despite Lexi being six years older than Joe, she grew up viewing Joe as a role model, particularly for his positive outlook on life. Joe was diagnosed with spinal muscular atrophy (SMA) Type 2 in 2002 when there
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Cure SMA Publishes Results from Survey Measuring Disease Burden in Adolescents and Young Adults
As treatment options increase for adolescents and young adults living with SMA, we need tools to measure the effectiveness of these treatments from the patient’s perspective. Dr. Chad Heatwole and his team at the University of Rochester have developed the Spinal Muscular Atrophy Health Index (SMA-HI) for this purpose.1 The SMA-HI is an SMA-specific survey