Latest News
AveXis Announces the Completion of Dosing of the Low-Dose Cohort in US Clinical Trial for Spinal Muscular Atrophy
AveXis, Inc., a clinic-stage gene therapy company, today announced the completion of dosing in the low dose cohort in the world’s first human gene therapy trial for the treatment of […]
Read More ›Isis Presents Phase II Trial Results
This Friday, October 10, Isis Pharmaceuticals will be presenting results from their Phase II clinical trials of ISIS-SMNRx in both children and infants. The results will be presented as part […]
Read More ›AveXis Receives Orphan Drug Designation
AveXis has received Orphan Drug Designation from the FDA for their gene therapy program, called chariSMA. Orphan Drug Designation is granted by the FDA to encourage and expedite the development […]
Read More ›Isis Begins Recruiting for Phase III Trial
Isis is currently recruiting participants for Endear, a pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The goal of this Phase III trial is to […]
Read More ›Cole P Randall Memorial Golf Tournament Raises $75,000
The Inaugural Cole P Randall Memorial Golf Tournament was held on Saturday, September 27, at The Reserve Vineyards and Golf Club in Portland, OR. This fun event had a full […]
Read More ›Cure SMA-Funded CALIBR Research Collaboration Expanded to USC
Cure SMA is pleased to announce that we have expanded our research collaboration with the California Institute for Biomedical Research (CALIBR) to include a collaborative effort with Dr. Chien-Ping Ko […]
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