On Sunday, May 26th, Regina Philipps will participate in the Vermont City Marathon and has already raised over $13,000 for Team Cure SMA while training. As an avid runner, Regina joined Team Cure SMA along with her sister, Amanda. They wanted to run the Vermont City Marathon with Team Cure SMA in honor of Regina’s […]
Team Cure SMA to Participate in Vermont City Marathon Read More »
The Ohio State University recently announced the 2019 honorees of the Distinguished Scholar Award. The award, established in 1978, recognizes exceptional scholarly accomplishments by senior professors who have compiled a substantial body of research, as well as younger faculty members who have demonstrated great scholarly potential. Dr. Arthur H.M. Burghes, PhD, Professor, Department of Biological
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The below statement is a community update from Scholar Rock on SRK-015. Dear SMA Community, Scholar Rock is dedicated to developing novel medicines to improve the lives of individuals who have Spinal Muscular Atrophy (SMA). We want to thank the SMA community for your continued support of our mission. We are excited to share that
Genentech, a member of the Roche Group, recently announced new data at the 71st American Academy of Neurology (AAN) Annual Meeting from the dose-finding Part 1 of the pivotal FIREFISH trial showing infants with Type 1 spinal muscular atrophy (SMA) achieved key motor milestones after one year of treatment with investigational risdiplam. Among the infants
AveXis, a Novartis company, recently announced interim data from ongoing trials of the investigational product Zolgensma® (onasemnogene abeparvovec-xioi; AVXS-101) that showed positive results across a broad spectrum of patients with spinal muscular atrophy (SMA). These included the first presentation of data from the Phase 1 STRONG trial, which showed motor function gains and milestone achievements
Cure SMA is thrilled to announce that we will be extending travel scholarship opportunities to adults with SMA (18 and over) who are attending the 2019 Annual SMA Conference, taking place June 28-July 1, 2019 at Disneyland Resort in Anaheim, CA. Thanks to a generous grant funded by Genentech, Cure SMA will be providing travel
Biogen today announced it will present new data focusing on SMA at the 71st annual meeting of the American Academy of Neurology (AAN) in Philadelphia, PA., May 4–11. Results from the NURTURE study demonstrate that pre-symptomatic infants with SMA treated with SPINRAZA over three years achieved motor milestones more consistent with normal childhood development. After
Biogen to Present Data at AAN Highlighting Spinraza and Emerging Biomarkers for SMA Read More »
Genentech, a member of the Roche Group, announced today that new data will be presented at the 71st American Academy of Neurology (AAN) Annual Meeting from May 4-10 in Philadelphia, PA. Presentations include data from a pivotal study for risdiplam in spinal muscular atrophy (SMA), which has the potential to become the first oral treatment
Genentech Presents New Data at the 2019 AAN Annual Meeting Read More »
Kyle and Laura Derkowski are co-chairs of the Cure SMA Virginia Chapter. The pair met during their first year in college through a mutual friend and were married in December 2014. The Derkowskis recently shared with Cure SMA their perspective on hope, community and leadership. At 18 months old, Kyle was diagnosed with spinal muscular
Community Spotlight: Kyle and Laura Derkowski Read More »
Biogen has announced it will present new data illustrating the rapidly progressive nature of spinal muscular atrophy (SMA) in adults, adolescents and older children at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida (April 13-17, 2019). Other Biogen presentations will highlight the benefits of pre-symptomatic treatment with data from the Nurture
Biogen Presents New Data Regarding Adults and Infants with SMA Read More »
