June 19, 2018
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 with Type 1 Spinal Muscular Atrophy...
June 16, 2018
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...
June 1, 2018
Scholar Rock, a biotechnology company based in Boston, recently announced that it has completed dosing of the first cohort and is continuing...
May 30, 2018
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 olesoxime, an investigational molecule in SMA.
Since we bought olesoxime from Trophos in 2015, we have had many...
May 24, 2018
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 patients with early infantile-onset SMA.”
SMA is caused by mutations in the SMN1 gene resulting in low levels of...
May 22, 2018
Biogen has released the following community statement on Spinraza access and new research data.
Dear Members of the SMA community,
Thank you for your continued support, efforts to raise awareness about SMA, and participation in clinical trials and studies as we seek to expand...
May 15, 2018
Dear SMA Community,
Following the announcement last month, we wanted to let you know that AveXis (the gene therapy company developing a new approach to treat SMA known as AVXS‐101) is
May 8, 2018
Cure SMA has awarded a $150,000 research grant to Chad Heatwole, MD, at the University of Rochester, for his project, " Development of a Clinically Relevant Outcome Measure for Pediatric SMA Therapeutic Trials.”
Dr. Heatwole and his team are working to develop SMA-specific...