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 »
Cure SMA recently launched the third annual Community Update Survey to address important issues in SMA treatment and care. The Community Survey covers several areas that are relevant to the real-world experiences of people living with spinal muscular atrophy (SMA). Lisa Belter is the Senior Research Data Analyst at Cure SMA. Every year, she and
Community Update Survey Q&A Read More »
The 2019 Annual SMA Conference is just over two months away and Cure SMA is excited to announce the details of the events held at this year’s conference. Open to all conference attendees, the popular Meet & Greet will be held on the evening of Friday, June 28th. This event includes the Researcher Relay Race
2019 Annual SMA Conference Events Announced Read More »
