Latest News
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 with Spinal Muscular Atrophy Type […]
Read More ›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 atrophy (SMA) who have completed […]
Read More ›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 CST (9:00am PST/10:00am MST/12:00pm EST). […]
Read More ›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 splicing modifier for SMA. The […]
Read More ›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 members of the SMA community, […]
Read More ›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 the Center for Gene Therapy […]
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