Latest News
AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age
AveXis, Inc., a Novartis company, today announced that they have received FDA approval for Zolgensma (formerly AVXS-101), a gene therapy that replaces the survival motor neuron 1 (SMN1) gene, which […]
Read More ›Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado
Audrey Lewis founded Families of SMA, now Cure SMA, 34 years ago. Audrey recognized early on the importance of attracting new and talented researchers to SMA, with the hope that […]
Read More ›Novartis Issues Community Statement on Branaplam Clinical Study
Dear SMA Community, We are writing to inform you that enrollment to the ongoing branaplam clinical study is now closed. Branaplam is currently being assessed for safety and efficacy in […]
Read More ›Team Biogen Runs from Chicago to Madison, Raising Over $12,000 for Cure SMA
On Friday, May 17th to Saturday, May 18th, Team Biogen will hit the ground running in the 2019 Reebok Ragnar Great Midwest, a 14-person relay race. The team’s goal is […]
Read More ›Cure SMA Publishes Journal Article on Benefit-Risk Preferences in SMA
Your voice has been heard! Cure SMA has published a paper, titled, Evaluating Benefit-Risk Decision-Making in Spinal Muscular Atrophy: A First-Ever Study to Assess Risk Tolerance in the SMA Patient Community […]
Read More ›Spring 2019 SMA Newborn Screening Update
We are thrilled to announce that Missouri, Pennsylvania and Vermont have now implemented permanent statewide SMA newborn screening, making 6 states that are now permanently screening statewide for SMA. Several […]
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