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 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
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
Cure SMA Care Center Network Site Expansion Read More »
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
Learn about Biogen’s latest news in SMA by viewing their Community Statement (available as a PDF) and a related video.
Biogen Shares Community Statement for SMA Awareness Month Read More »
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,
FDA Issues Safety Warning Regarding Neck Float Devices for Babies Read More »
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
Community Spotlight: Alexandra and Joe Lakhman Read More »
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
The recent availability of new treatments for SMA would not have been possible without the participation of SMA-affected individuals in clinical trials. Yet participating in clinical trials can be stressful. We at Cure SMA want to understand how future trials can be designed to reduce stress for participants and their parents and caregivers. To achieve
In June, SMA researchers and clinicians from around the world met in Anaheim, CA, for the 2022 Annual SMA Research and Clinical Care Meeting. There they shared their most recent data with the goal of accelerating the pace of research into SMA treatments and achieving excellence in SMA clinical care. Below is a summary of
2022 Cure SMA Annual Research Meeting Summaries – Special and Clinical Research Session Read More »
