Research
Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers
Scholar Rock today announced positive final top-line results from the Phase 1 clinical trial of its product candidate, SRK-015, a highly specific inhibitor of myostatin […]
Read More ›AveXis and Cure SMA to Co-Host Webinar on the FDA’s Approval of Zolgensma
On Wednesday, June 12th, at 10am CDT (8am PDT/9am MDT/11am EDT) AveXis, a Novartis company, and Cure SMA will co-host a webinar on the FDA […]
Read More ›AveXis Issues Community Statement on Zolgensma Approval
Dear SMA Community, We have exciting news to share with you — a major milestone has been reached in the fight against spinal muscular atrophy […]
Read More ›AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age
AveXis, Inc., a Novartis company, today announced that they have received FDA approval for Zolgensma (formerly AVXS-101), a gene therapy that replaces the survival motor […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
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 […]
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 […]
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 […]
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