Advocacy
6th Annual Congressional Dinner Furthers SMA Advocacy Work
This year’s 6th Annual “Hope on the Hill” Congressional Dinner will be held on Tuesday, November 29, bringing together families, government, and industry for an […]
Read More ›SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA
Cure SMA is excited to announce that the SMA community has been granted a Patient Focused Drug Development Meeting with the FDA. At the Patient […]
Read More ›Cure SMA Announces Newborn Screening Initiative in Partnership with MDA
Twice this year, Cure SMA has had the opportunity to testify before the federal Advisory Committee on Heritable Disorders on the need for newborn screening […]
Read More ›We’re Partnering With The Mighty!
We’re thrilled to announce a new partnership that will bring Cure SMA’s resources in front of The Mighty‘s wide-reaching readership. We will now have a […]
Read More ›Cure SMA Plans a Series of Educational Modules for the SMA Community
With the recent news that Biogen and Ionis will submit nusinersen for FDA approval—along with other recent developments, including the breakthrough designation for AveXis’s gene […]
Read More ›Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy
Dr. Chris Lorson and his colleagues have published a paper, “Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy,” in […]
Read More ›Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports
Dr. Rashmi Kothary and his team have recently published a paper, “Differential induction of muscle atrophy pathways in two mouse models of spinal muscular atrophy” […]
Read More ›Cure SMA Posts Keynote Presentations on the NDA Process
The ultimate goal in drug development is to get treatments approved for as many people as possible, as quickly as possible. Because these issues are […]
Read More ›2016 Update on SMA-FDA Interactions
The SMA drug pipeline has grown dramatically in just over a decade. Of the 18 programs in the pipeline, six of these are in clinical […]
Read More ›Cure SMA and the SMA Community Featured by Astellas Pharmaceuticals
Cure SMA is featured by Astellas Pharmaceuticals as part of their new Changing Tomorrow Together website. Cure SMA Research Director Jill Jarecki contributed a spotlight […]
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