For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes and challenges. We know that the voice of our community is powerful. By sharing our stories, we can communicate our priorities to the FDA and regulators, provide insight into daily life with SMA and the ongoing […]
Biogen presented new data from the Phase 3 ENDEAR study of SPINRAZA™ (nusinersen), which demonstrated a statistically significant reduction in the risk of death or permanent ventilation in SPINRAZA-treated infants with spinal muscular atrophy (SMA), compared to untreated infants. The data was presented at the British Pediatric Neurology Association (BPNA) annual conference in Cambridge, UK,
With the news today that the first-ever patient has received the first commercial Spinraza dose, we are releasing an updated version of our SMA drug pipeline. This new version reflects the approval of Spinraza, as well as the true start of a key stage to ensure that more and more patients are able to gain
Updated SMA Drug Pipeline Released as First Commercial Patient Receives Spinraza Read More »
Tuesday, April 18, 2017, has been announced as the date for the SMA Patient-Focused Drug Development Meeting with the FDA. At the Patient Focused Drug Development (PFDD) Meeting, individuals and families from throughout our community will have the opportunity to share their stories with the FDA on a variety of topics important to our community.
SMA Patient-Focused Drug Development Meeting with FDA Set for April 18, 2017 Read More »
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 tangible support for families affected by SMA, easing the difficulties of an SMA diagnosis, and showing each family that they are not alone and that there is hope. Our family
Cure SMA Assists Hundreds of Families in 2016 Read More »
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 recieve Cure SMA funding often publish their findings in peer-reviewed journals. Published articles allow the experiments and results to be reviewed and vetted by other scientists, who may then use
Cure SMA Funding Results in 16 New Research Publications Read More »
This has been an historic year for the SMA community. On December 23, the FDA announced its approval of Spinraza, the first-ever approved treatment for SMA. In addition to the approval of Spinraza, in 2016 the SMA drug pipeline reached notable highs. There are now: 18 active programs 6 programs in clinical trials 14 pharmaceutical
2016 Research Year-in-Review: An Historic Year for Our Community Read More »
