Carrie Menke is a South Dakota native who resides in Sioux Falls with her husband Tony and their four children — Xavier, Colette, Avila, and Rose. Carrie is one of the many advocates across the country who is supporting efforts to have spinal muscular atrophy (SMA) added to her state’s newborn screening panel. Her passion […]
Community Spotlight: Xavier and Rose’s SMA Diagnosis Read More »
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 with spinal muscular atrophy (SMA) and we’d like to share an update with you. (Please see the press release issue by Novartis
Team Biogen hit the ground running throughout the month of August in honor of SMA Awareness Month. Together, the team logged 22,059 miles collectively from August 1st to August 31st. Nearly 300 Biogen employees came together, virtually of course, to raise funds and awareness for SMA. They successfully raised close to
Team Biogen Runs Over 22,000 Miles During SMA Awareness Month Read More »
September is Newborn Screening Awareness Month, a chance to talk about the progress we have made in getting spinal muscular atrophy (SMA) added to newborn screening panels and the work still to be done. This month, you will hear about newborn screening milestones, stories, and steps you can take to ensure all states screen newborns
Community Spotlight: Braedon’s Newborn Screening Diagnosis Read More »
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 Biogen’s latest efforts on the following: We are committed to sharing information that will aid healthcare providers and individuals with SMA in
Biogen Q3 2020 Community Statement: COVID-19 Updates and Latest Milestones Read More »
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 granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular Atrophy (SMA), a progressive, rare genetic disease that
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 shared updates and members of the SMA community shared public comments. Nicole Stickane shared her family’s experience in support of adding SMA
Community Spotlight: Nicole Stickane Advocates for Newborn Screening in Texas Read More »
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 milestone. Today, the U.S. Food and Drug Administration (FDA) has approved risdiplam for the treatment of spinal muscular atrophy (SMA) in adults
Genentech Releases Evrysdi™ FDA Approval Letter to the SMA Community Read More »
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 muscular atrophy (SMA). Evrysdi showed clinically meaningful improvements in motor function across two clinical trials in people with varying ages and levels
Genentech Receives FDA Approval of Evrysdi (risdiplam) for the Treatment of SMA Read More »
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 our current support programs, Cure SMA is thrilled to launch a brand-new program offered to all affected individuals with SMA to help
Cure SMA Launches New Medical Alert Bracelet Support Program Read More »
