Latest News
Spring 2019 SMA Newborn Screening Update
We are thrilled to announce that Missouri, Pennsylvania and Vermont have now implemented permanent statewide SMA newborn screening, making 6 states that are now permanently screening statewide for SMA. Several […]
Read More ›Team Cure SMA to Participate in Vermont City Marathon
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 […]
Read More ›Dr. Arthur Burghes Named 2019 Distinguished Scholar
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 […]
Read More ›Scholar Rock Issues Community Statement on the Initiation of a New Clinical Trial for SRK-015
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 […]
Read More ›Genentech Presents Data from the Risdiplam Pivotal FIREFISH and SUNFISH Studies in Spinal Muscular Atrophy at the AAN Annual Meeting
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 […]
Read More ›AveXis Presents Data at AAN Annual Meeting Demonstrating Efficacy of Zolgensma in Broad Spectrum of Spinal Muscular Atrophy (SMA) Patients
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 […]
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