As we continue to quarantine and see a new wave of COVID-19 cases across the country, Cure SMA would like you to know that our work for the spinal muscular atrophy (SMA) community continues. We remain vigilant in our monitoring of the pandemic statistics and responding to the needs of the entire SMA community. Your […]
Now Available! New COVID-19 PPE Package from Cure SMA Read More »
Each year since 2017, Cure SMA has conducted a Community Update Survey. This online questionnaire is sent to all individuals with spinal muscular atrophy (SMA) and caregivers of children with SMA in the Cure SMA database to capture self- and caregiver-reported data on disease characteristics and health outcomes. Cure SMA uses data collected through the
Results from Annual SMA Community Update Survey Now Published Read More »
A little more than six months ago, Cure SMA launched the Summit of Strength Virtual Webinar Series , a program designed to help support individuals and families with SMA manage the uncertainty of life in quarantine during the global COVID-19 pandemic. We have now hosted 13 Summit of Strength educational webinars and are coming to
Summit of Strength Virtual Webinar Series Continues Its Success! Read More »
Through Cure SMA’s partnership with NeurologyLive, a new patient-centered article, “Early Intervention in SMA: How Access to Therapy Will Alter Disease Management As We Know It,” is now available for viewing online. This piece features an interview with Dr. Mary Schroth, Chief Medical Officer at Cure SMA, and discusses successes in newborn screening
Cure SMA has published a paper, titled “Spinal Muscular Atrophy and COVID-19: Guidance and Resources for Providers,” in partnership with NeurologyLive. The article highlights findings from a recent survey conducted by Cure SMA to measure the impact of COVID-19 on the spinal muscular atrophy (SMA) patient and family community. As of June 1,
Cure SMA Publishes Paper in Partnership with NeurologyLive on Impact of COVID-19 Read More »
Novartis Gene Therapies today announced new interim data from the ongoing Phase 3 STR1VE-EU clinical trial for Zolgensma® (onasemnogene abeparvovec). The data demonstrates that patients with spinal muscular atrophy (SMA) Type 1 continued to experience significant therapeutic benefit, including event-free survival, rapid and sustained improvement in motor function, and motor milestone achievement, including
Novartis Presents New Zolgensma® Data Further Demonstrating Therapeutic Benefit Read More »
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 »
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
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 »
