Dear SMA Community, We recently received important feedback from the U.S. Food and Drug Administration (FDA) following their review of data from the STRONG study of AVXS-101 intrathecal (IT) formulation in older patients ...
Despite the new reality of postponed events, remote working, and social distancing, Cure SMA would like you to know that our work for the SMA community continues. Please stay ...
Genentech, a member of the Roche Group, today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Evrysdi (risdiplam) to treat adults and children 2 months and older with spinal ...
We are excited to share with the SMA community that the U.S. Food and Drug Administration (FDA) has granted Cure SMA a Critical Path Innovation Meeting (CPIM). This means that key leaders at ...
Biogen Inc., today announced, it plans to initiate a global Phase 4 clinical study, RESPOND, to examine the clinical benefit and assess the safety of SPINRAZA (nusinersen) in infants and children with ...
Ben Lou is a 17-year-old from San Diego who is fluent in both English and Chinese. He is a math whiz and fascinated by science. Ben also has had total body weakness from ...
Cure SMA would like to thank everyone who attended last week’s 2020 Virtual SMA Conference! It was an impactful week of online workshops, networking, and community. We are happy to announce that there ...
Genentech, a member of the Roche Group, shared two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular ...
Biogen, Inc., today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA), which is transforming expectations of early treatment with SPINRAZA (nusinersen). In ...
Cure SMA is pleased to announce that the 2020 Virtual SMA Conference booklet is now available for download. The booklet includes important details on the Virtual SMA Conference, including: A full agenda with ...
In the midst of the ongoing Coronavirus (COVID-19) global pandemic, Cure SMA is excited to announce its new funding for the Pediatric Neuromuscular Clinical Research Network (PNCRN). In 2020, Cure SMA will provide ...
Cure SMA is proud to share with the SMA community that it has published a paper in the Orphanet Journal of Rare Diseases, titled ‘The SMA Clinical Trial Readiness Program: Creation ...
