Cure SMA Welcomes New Care Center Network Site!
We'd like to give a warm welcome to Children’s Hospital Colorado as a newly added Cure SMA Care Center! Thank you to Loree, Ward and Lyza Weisman for your generous donation supporting your ...
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 ...
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 ...
Cure SMA Publishes Research Exploring What Is Important to SMA Clinical Trial Participants
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 ...
Financial Planning with a Disability
When you first learned that you or your child had SMA, you probably began analyzing your home life, your or your child’s education, and other immediate concerns. In time, you realized that the ...
Community Spotlight: Nikki McIntosh
Nikki McIntosh is known as the founder of Rare Mamas, an online resource that supports and empowers mothers who have children with rare diseases. She and her husband, Tony, live in Southern California ...
Community Spotlight: Scurria Family
John and Krista Scurria, of Baton Rouge, La., believe in a parent’s intuition. Krista first became worried about the development of her oldest child, Josh, while she was still pregnant with him. But ...
2019 SMA Researcher Meeting Summary: Clinical Drug Development
The annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year, we had a record setting 735 attendees join together in Anaheim, CA. The goal ...
What to Expect in a Clinical Trial
Clinical trials for spinal muscular atrophy (SMA) help researchers answer important questions about the disease and investigational drugs, providing information that may help the development of future medical treatments. Although there is now ...
Cure SMA Statement on Tax Reform Bill and the Repeal of the Individual Mandate
On Wednesday, Congress passed the most sweeping tax bill in a generation. Cure SMA has been monitoring and advocating on your behalf as this legislation went through the Congress. We are happy to ...
Cure SMA Announces $5.05 Million in Research Funding for FY18
Over the past fiscal year—from July 1, 2016, to June 30, 2017—Cure SMA has funded over $2.5 million in new research funding. This funding will be used strategically to help accelerate research, and ...
Biogen Completes Rolling Submission of New Drug Application to FDA
Biogen and Ionis today announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational ...