Research
Dr. John Kissel, Chair of Neurology at OSU Wexner Medical Center, Retires After 40 Years
Dr. John Kissel has treated patients with neuromuscular disorders from all over the United States. He specializes in spinal muscular atrophy and has been instrumental […]
Roche Releases Community Update on Risdiplam
The following is a community update from Genentech/Roche on the risdiplam (RG7916) program. Dear SMA advocacy community, As requested, we are very pleased to share […]
Cure SMA Awards $300,000 Grant to Umrao Monani, PhD, Columbia University
Cure SMA has awarded a $300,000 preclinical drug discovery grant to Umrao Monani, PhD, at Columbia University, for his project, ” Restoring function at the […]
Summer 2018 Issue of Compass Now Available
The summer 2018 issue of Compass is now available online. As part of our most recent round of funding, we’ve announced ten grants totaling $1,325,000 […]
PTC Therapeutics Announces Risdiplam (RG7916) is Well Tolerated at All Dose Levels With No Drug-Related Safety Findings
On Saturday, PTC Therapeutics, Inc. announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies […]
Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference
Today, Cytokinetics announced data from the Phase 2 clinical study of reldesemtiv in patients with SMA, presented in an oral presentation by John W. Day, […]
Biogen to Present New Data at the 2018 Annual SMA Conference
Yesterday, Biogen announced it will present data from its SPINRAZA clinical development program –the largest of its kind, with more than six years of data–for […]
Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015
Scholar Rock, a biotechnology company based in Boston, recently announced that it has completed dosing of the first cohort and is continuing enrollment of healthy […]
Roche Releases Community Statement on Olesoxime Program
Roche today provided the following community statement on olesoxime. Dear members of the SMA Community, We would like to provide you with an update about […]
Cure SMA Awards $150,000 Grant to Kathryn Swoboda, MD, Massachusetts General Hospital
Cure SMA has awarded a $150,000 research grant to Kathryn Swoboda, MD, at the Massachusetts General Hospital, for her project, “Clinical and pathologic correlations in […]