Dear Members of the SMA Community, We continue to work with the healthcare community to help people and families living with SMA get the support they need. As such, we wanted to share ...
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 ...
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has ...
In late July, the Texas Newborn Screening Advisory Committee held a meeting— virtually, of course —to hear an update on the status of statewide newborn screening for spinal muscular atrophy (SMA). State officials ...
Dear SMA Patient Advocacy Community, As part of our ongoing partnership and following your request to receive updates about the risdiplam clinical development program, we are delighted to share with you a much-anticipated ...
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 ...
Along with funding SMA research and care, Cure SMA provides thousands of individuals and families with vital support and resources that help people navigate life with spinal muscular atrophy (SMA). In addition to ...
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 ...
July 2020 marks the 2-year anniversary of when the U.S. Secretary of Health and Human Services recommended that SMA be added to state newborn screening panels. This month, you’ll hear about newborn screening ...
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 ...
