Our Impact
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
FDA Approves Spinraza for SMA
On December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA. […]
Cure SMA Participates in a Special FDA Listening Session
Last week, Cure SMA participated in a listening session with FDA Commissioner Robert M. Califf, MD, MACC. The FDA organized the listening session in order […]
Cure SMA Announces Additional Conference Support for Newly Diagnosed Families
Thanks to the generous funding provided by the Erin Trainor Memorial Fund, Cure SMA is now offering additional support for newly diagnosed families to attend […]