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 […]
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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
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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
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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
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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.
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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
Cure SMA is pleased to announce that we are accepting grant applications for funding of research projects and postdoctoral fellowships, under a competitive review by our Scientific Advisory Board (SAB). Please click here to view the SAB roster. As we are now able to return to in-person activities for our SMA community, including conferences and
Cure SMA is pleased to announce the launch of an expanded Phase 7 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, SMA Europe, Cure SMA, and other nonprofit organizations, to share information, ideas, and data to benefit the broader SMA community. Through the Industry Collaboration,
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