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 is missing or mutated in individuals with SMA. This first approval is for intravenous (IV) delivery of gene therapy. The FDA has approved Zolgensma for […]
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 they would commit their careers to developing a treatment and cure for SMA. Cure SMA honors Audrey’s legacy with the Audrey Lewis Young Investigator Award,
Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado Read More »
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 a Phase 1/2 clinical study in Type 1 infantile-onset SMA. We are pleased to announce that we have enrolled 25 infants into Part 2 of
Novartis Issues Community Statement on Branaplam Clinical Study Read More »
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 to raise funds and awareness for the SMA community. Team Biogen has already raised over $12,000 for Team Cure SMA while training for this epic
Team Biogen Runs from Chicago to Madison, Raising Over $12,000 for Cure SMA Read More »
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 in the Journal of Clinical Therapeutics. Our community’s perceptions of benefit-risk are essential to the evaluation of potential therapies for SMA. To bring this critical
Cure SMA Publishes Journal Article on Benefit-Risk Preferences in SMA Read More »
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 other states have adopted SMA newborn screening and have moved one step closer to implementing statewide screening. There are now 4 states running SMA screening
Spring 2019 SMA Newborn Screening Update Read More »
On Sunday, May 26th, Regina Philipps will participate in the Vermont City Marathon and has already raised over $13,000 for Team Cure SMA while training. As an avid runner, Regina joined Team Cure SMA along with her sister, Amanda. They wanted to run the Vermont City Marathon with Team Cure SMA in honor of Regina’s
Team Cure SMA to Participate in Vermont City Marathon Read More »
The Ohio State University recently announced the 2019 honorees of the Distinguished Scholar Award. The award, established in 1978, recognizes exceptional scholarly accomplishments by senior professors who have compiled a substantial body of research, as well as younger faculty members who have demonstrated great scholarly potential. Dr. Arthur H.M. Burghes, PhD, Professor, Department of Biological
Dr. Arthur Burghes Named 2019 Distinguished Scholar Read More »
The below statement is a community update from Scholar Rock on SRK-015. Dear SMA Community, Scholar Rock is dedicated to developing novel medicines to improve the lives of individuals who have Spinal Muscular Atrophy (SMA). We want to thank the SMA community for your continued support of our mission. We are excited to share that
Genentech, a member of the Roche Group, recently announced new data at the 71st American Academy of Neurology (AAN) Annual Meeting from the dose-finding Part 1 of the pivotal FIREFISH trial showing infants with Type 1 spinal muscular atrophy (SMA) achieved key motor milestones after one year of treatment with investigational risdiplam. Among the infants
