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 6-month interim results from their clinical program for its product candidate SRK-015, a treatment being developed for spinal muscular atrophy (SMA). SRK-015 is a highly selective inhibitor of latent myostatin and […]
October is National Disability Employment Awareness Month, with an official theme of Increasing Access and Opportunity. Throughout the month, we have been posting stories about adults with SMA in the workforce and are pleased to share this perspective column from Spencer Cook, as seen in the latest Fall issue of Directions. Living with a disability in the
Community Spotlight: Perspectives from Spencer Cook, Registered Accessibility Specialist Read More »
October is National Disability Employment Awareness Month, with an official theme of Increasing Access and Opportunity. Throughout the month, we will be posting stories about adults with SMA in the workforce and are pleased to share this story about Kayley Shade. Kayley Shade is a 28-year-old sports fan from Hartford, S.D. She has lived with spinal muscular
Community Spotlight: Kayley Shade of Sioux Falls Storm 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
October is National Disability Employment Awareness Month, with an official theme of Increasing Access and Opportunity. Throughout the month, we will be posting stories about adults with SMA in the workforce and are pleased to share this story about AJ Brockman, 32, of Palm Beach Gardens, FL. AJ Brockman says he wears many hats in life. No
Community Spotlight: AJ Brockman of Brewhouse Gallery and Kelsey City Brewing Company Read More »
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 »
Genentech, a member of the Roche Group, today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi™ (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve
Genentech Presents New 2-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA Read More »
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
