Our Impact
Cure SMA Contributes Article to a Special Issue of the Journal Gene Therapy
To celebrate the FDA approval of Spinraza, Cure SMA contributed an article to the September special issue of the journal Gene Therapy. The issue , […]
Read More ›Centers for Medicare and Medicaid Services (CMS) Survey
On August 3rd, as part of multiple days of advocacy visits, Cure SMA staff met with leaders from the Medicare division of the Centers for […]
Read More ›SMA Families and Cure SMA Staff Visit Capitol Hill for Advocacy Efforts
On August 2nd, members of the spinal muscular atrophy community kicked off SMA Awareness Month by meeting with a dozen Senate and House congressional offices […]
Read More ›RUSP Nomination for SMA Accepted into Evidence Review
The Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (SACHDNC) announced that they have accepted spinal muscular atrophy into the review process for […]
Read More ›SMA Community’s Voice Heard “Loud and Clear” at Last Week’s Patient Focused Drug Development Meeting with the FDA
On April 18, 2017, the SMA community—families, clinicians, researchers, industry and regulators—gathered for a Patient-Focused Drug Development (PFDD) Meeting with the FDA. As part of […]
Read More ›Cure SMA Awards $140,000 Grant to Arthur Burghes, PhD, The Ohio State University
Cure SMA has awarded a $140,000 research grant to Arthur Burghes, PhD, at The Ohio State University, for his project, “Identification of SMA modifiers and […]
Read More ›SMA is Submitted to the Federal Newborn Recommended Uniform Screening Panel (RUSP)
Cure SMA and the SMA Newborn Screening Coalition are excited to announce we have completed the submission of SMA to the Recommended Uniform Screening Panel […]
Read More ›Registration Opens for the SMA Patient-Focused Drug Development Meeting with the FDA
Registration is now open for the SMA Patient-Focused Drug Development (PFDD) meeting with the FDA. The PFDD meeting will be held at the College Park […]
Read More ›Cure SMA Assists Hundreds of Families in 2016
Each year, Cure SMA provides thousands of families with vital support and resources to help them live active, engaged and hopeful lives. Our programs provide […]
Read More ›Cure SMA Funding Results in 16 New Research Publications
An important goal of our research funding strategy is to share scientific findings with the broader scientific community. To help accomplish this goal, scientists who […]
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