Front Page News
Zolgensma Research Updates from Novartis Gene Therapies
New Zolgensma data demonstrate age-appropriate development when used early, real-world benefit in older children, and durability 5+ years post-treatment Novartis Gene Therapies recently shared new […]
Read More ›Cure SMA Publishes Quality of Life in Teens and Young Adults Manuscript in Orphanet Journal of Rare Diseases
Current knowledge regarding clinical meaningfulness and quality of life amongst teens and young adults with spinal muscular atrophy (SMA) is limited. Much of the available […]
Read More ›Biogen SMA Q1 2021 Community Statement
Dear Members of the SMA Community, Each year, the entire rare disease community comes together on the last day of February to […]
Read More ›Community Spotlight: I am…Viola Dwyer
February 28 is Rare Disease Day. Throughout the month—in recognition of our Redefining Rare initiative—we have been posting stories about SMA community members, showcasing their […]
Read More ›Jessica’s Story: Remembering Piper Grace
February 28 is Rare Disease Day. Throughout the month—in recognition of our Redefining Rare initiative—we will be posting stories about SMA community members, showcasing their […]
Read More ›Results from Genentech’s Evrysdi Study in Infants with Type 1 SMA Published in NEJM
Genentech, a member of the Roche Group, announced in a press release that Evrysdi™ (risdiplam) data from the dose finding Part 1 of the pivotal […]
Read More ›Cure SMA Launches Virtual Therapy Program for Adults with SMA
Adults with spinal muscular atrophy (SMA) are invited to take advantage of Cure SMA’s latest support resource, the Adults with SMA Virtual Therapy Program, which […]
Read More ›Community Spotlight: I Am…Kelsey Roberts
February 28 is Rare Disease Day. Throughout the month—in recognition of our Redefining Rare initiative — we will be posting stories about SMA community members, […]
Read More ›Cure SMA Coronavirus (COVID-19) Information Center
Over the past year and a half, we hope that you have found this page to be a helpful resource as you navigated the COVID-19 […]
Read More ›Cure SMA Career Panel Webinar Series to Launch in February
Cure SMA is excited to invite all members of the SMA community to join our new, three-part Career Panel Webinar Series, generously sponsored by Biogen. […]
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