Front Page News
Cure SMA Welcomes the North Jersey Chapter
Cure SMA is pleased to announce the creation of a new chapter in northern New Jersey, serving families and supporters with zip codes starting with […]
Cure SMA and Other Venture Philanthropy Leaders Share Insight at Partnering for Cures Panel
Cure SMA President Kenneth Hobby was a featured panelist at Partnering for Cures, a conference convened by the Milken Institute’s FasterCures initiative. At the event, […]
Cytokinetics Announces New Details on Upcoming Phase 2 Trial
Cytokinetics, in collaboration with Astellas, has announced further details about a planned Phase 2 clinical trial of CK-2127107 in patients with spinal muscular atrophy (SMA). […]
Cure SMA Publishes Journal Article on the Patient Voice
Cure SMA, along with collaborators from Biogen and the SMA Foundation, have published a paper in the journal BMC Neurology. Over the past year, we’ve […]
Cure SMA-Funded Researchers Publish Paper in Journal Pediatrics
Cure SMA-funded researchers Rebecca Hurst Davis and Kathy Swoboda have published a paper, “Responses to Fasting and Glucose Loading in a Cohort of Well Children […]
Isis Pharmaceuticals Announces SHINE, an Open-Label Extension Study
Isis Pharmaceuticals, Inc. announced that it has initiated an open-label extension study, SHINE. The SHINE study provides ISIS-SMNRx to infants and children with spinal muscular […]
Cure SMA to Host Clinical and Regulatory Webinar
On Monday, November 9, Cure SMA will be hosting a one-hour webinar on clinical trials and regulatory topics. The webinar will be held at 11:00am […]
PTC Announces New Clinical Trial Results at World Muscle Congress
PTC Therapeutics, in partnership with Roche and the SMA Foundation, recently announced new results from their ongoing Phase 2 trial of RG7800, a small molecule […]
Community Update on ISIS-SMNRx from Biogen and Isis
Biogen and Isis Pharmaceuticals recently provided an update on the clinical development of ISIS-SMNRx, which is currently being tested in Phase 3 clinical trials: Dear […]
New Gene Therapy Results Announced at World Muscle Congress
Nationwide Children’s Hospital/AveXis recently announced new results from their ongoing Phase 1 trial of systemic gene therapy for SMA. Dr. Jerry R. Mendell, Director of […]