Advocacy
Help Us Shape the Future Together, and Reach our Goal of $750,000
This has been an historic year for the SMA community. On October 28, our pharmaceutical partners at Biogen and Ionis announced that their New Drug […]
Read More ›Cure SMA Launches Insurance Coverage and Payment Policy Project
Earlier this year, Cure SMA initiated an insurance coverage and payment policy project, in partnership with District Policy Group. The goal of this project is […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
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