Research
2018 SMA Researcher Meeting Summary: SMA Therapy Development
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA and seeks to create open communication of early, unpublished […]
Read More ›2018 SMA Researcher Meeting Summary: Clinical Research Studies for SMA
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA and seeks to create open communication of early, unpublished […]
Read More ›2018 SMA Researcher Meeting Summary: Basic Research Podium Presentations
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA and seeks to create open communication of early, unpublished […]
Read More ›2018 SMA Researcher Meeting Special Session Summary: Clinical and Basic Questions of SMA in the Era of SMN Repletion: Challenge and Opportunity
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA and seeks to create open communication of early, unpublished […]
Read More ›Cure SMA Awards $300,000 Grant to Kevin Hodgetts, PhD, The Brigham and Women’s Hospital
Cure SMA has awarded a $300,000 preclinical drug discovery grant to Kevin Hodgetts, PhD, at the Brigham and Women’s Hospital, for his project, ” Pre-Clinical […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
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