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 ...
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 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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 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 ...
