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 – Basic Research Sessions Read More »
Congress has recognized the unmet and new needs of individuals with SMA by including legislative language in must-pass legislation asking the National Institutes of Health (NIH) to continue research in SMA. At the 2022 Cure SMA conference and throughout the summer, individuals with SMA and their families educated Congress about existing challenges faced by the
Cure SMA Advocacy Campaign Leads to Congressional Support for More SMA Research Read More »
On Thursday, August 4, 2022, six members of the SMA community will have the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) about their lived experiences with SMA and their unmet medical needs. This Patient-Led Listening Session will be a small, private teleconference attended by patient advocates, FDA representatives from the
Cure SMA to Hold Patient-Led Listening Session with FDA Read More »
Cure SMA is pleased to announce the launch of our newly updated merch store! The merch store features all the most popular Cure SMA t-shirts, hats, zip-up jackets, and more, as well as new items, which include: Black Performance Full-Zip Fleece Bucket Hat Women’s White Reflective Performance Half Zip Women’s Performance Racerback Tank In addition
Shop New Cure Merch at Our Updated Online Store Read More »
Thank you to everyone who attended the 2022 Annual SMA Conference at the Disneyland Hotel in Anaheim, California! It was an impactful weekend of opportunities to connect, learn, and have fun! We are happy to announce that, including on-site registration, there were over 2,100 attendees in total, which was a record for the Annual SMA
Thank You for a Wonderful 2022 Annual SMA Conference! Read More »
Cure SMA is pleased to announce the participation of scientific leadership in the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 13–16, 2022), the American Academy of Neurology (AAN) 2022 Annual Meeting (April 2–7, 2022, and the upcoming Cure SMA Research & Clinical Care Meeting (June 15–17, 2022). Our poster presentations feature data from research
Cure SMA is pleased to announce the launch of its first annual State of SMA report. The purpose of this report is to share highlights from Cure SMA’s three databases: the membership database with patient reported outcomes on over 9700 affected individuals worldwide that also includes data from our annual community update survey, the SMA
Check Out Cure SMA’s State of SMA Report Read More »
With today’s newborn screening action by Oregon and New Mexico, Cure SMA has reached a major screening milestone for spinal muscular atrophy (SMA), with 97 percent of all U.S. newborns screened for SMA. Oregon and New Mexico confirmed with Cure SMA that babies born today in their states will now be screening for SMA, making
We are pleased to share a community update on Novartis Gene Therapies’ Clinical Trial Program.
SMA Community Update from Novartis Gene Therapies Read More »
Genentech, a member of the Roche Group, today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to include babies under two months old with SMA. The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of
