Our Impact
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 […]
Read More ›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. […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
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