Front Page News
Together, We Can Cure SMA
Cure SMA is our name, and it’s also our rallying cry. It’s why we keep funding research and family support. And we know it’s why […]
Read More ›Promising Results Published on Cure SMA-Funded Gene Therapy Project
A manuscript from the laboratory of Dr. Brian Kaspar of Nationwide Children’s Hospital was recently published in the journal Molecular Therapy. The paper, “Improving single […]
Read More ›Read the Fall 2014 Issue of Compass
The latest issue of Compass, our research publication, is now available online. This Fall 2014 issue includes news from our 2014 SMA researcher meeting, and […]
Read More ›Cure SMA Honors Scott Geller with 1st Annual Community Partnership Award
We are proud to announce Scott Geller, who chaired the Chesapeake Chapter Golf Outing for fourteen years, as the first ever recipient of the Annual […]
Read More ›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 […]
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