Front Page News
Isis Releases Phase II Clinical Trial Results
Today, Isis released results from their Phase II open-label clinical trials of ISIS-SMNRx. These results covered two separate trials: a trial in infants with SMA […]
Read More ›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 […]
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 […]
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 […]
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 […]
Read More ›Cure SMA and Isis Announce Holiday Card Contest TEST
It’s only September, but the holidays are right around the corner! Cure SMA and Isis Pharmaceuticals are teaming up for a holiday card contest. This […]
Read More ›Cure SMA and Isis Announce Holiday Card Contest
It’s only September, but the holidays are right around the corner! Cure SMA and Isis Pharmaceuticals are teaming up for a holiday card contest. This […]
Read More ›Cure SMA and PPMD Invite Spinal Muscular Atrophy Parents to Take Part in Two Clinical Trial Surveys
Cure SMA and Parent Project Muscular Dystrophy (PPMD) are conducting two different surveys about clinical trial participation. The goal is to find out more about […]
Read More ›Cure SMA Featured in Assay and Drug Development Technologies
Cure SMA was featured in the July/August issue of Assay and Drug Development Technologies, a scientific journal that focuses on the development of new drugs. […]
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