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 their participation in the Phase 3 ENDEAR and CHERISH studies and are eligible to participate in SHINE. All patients in the SHINE study will receive […]

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 presentation was made at last week’s World Muscle Congress in the United Kingdom. Results from the first cohort of patients enrolled in the Phase 2

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, Over the past few months, we have received many questions from members of the SMA community about ISIS-SMNRx and when it might be widely available

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 at The Research Institute at Nationwide Children’s Hospital (NCH), made the presentation at last week’s World Muscle Congress in the United Kingdom. Dr. Mendell, lead