Front Page News
Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado
Audrey Lewis founded Families of SMA, now Cure SMA, 34 years ago. Audrey recognized early on the importance of attracting new and talented researchers to […]
Novartis Issues Community Statement on Branaplam Clinical Study
Dear SMA Community, We are writing to inform you that enrollment to the ongoing branaplam clinical study is now closed. Branaplam is currently being assessed […]
Team Biogen Runs from Chicago to Madison, Raising Over $12,000 for Cure SMA
On Friday, May 17th to Saturday, May 18th, Team Biogen will hit the ground running in the 2019 Reebok Ragnar Great Midwest, a 14-person relay […]
Cure SMA Publishes Journal Article on Benefit-Risk Preferences in SMA
Your voice has been heard! Cure SMA has published a paper, titled, Evaluating Benefit-Risk Decision-Making in Spinal Muscular Atrophy: A First-Ever Study to Assess Risk Tolerance […]
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 […]
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. […]
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 […]
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 […]
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 […]
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 […]