Latest News
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 […]
Read More ›Registration Open for the 2016 SMA Researcher Meeting
Registration is now open for industry and academic researchers to attend our 2016 SMA Researcher Meeting, which will be held June 16-18 in Anaheim, CA, at the Disneyland Hotel. The […]
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