Advocacy
Cure SMA Joins SMA Europe and TREAT-NMD for Meeting with EMA Regulators
Cure SMA will be participating in a one-day workshop with The European Medicines Agency (EMA—the European equivalent of the FDA), SMA Europe and TREAT-NMD to […]
Cure SMA to Host Webinar on the New Drug Application Process
On Tuesday, December 6, Cure SMA will be hosting a one-hour webinar on the New Drug Application (NDA) process. The webinar will be held at […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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” […]
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 […]