Front Page News
Southern California Gala of Hope Brings Community and Industry Partners Together to Raise $80,000
On November 15, 140 guests gathered for the Inaugural Southern California Cure SMA Gala of Hope. This fantastic evening raised over $80,000 in support of […]
Read More ›Cure SMA Scientific Advisory Board Meets in Washington D.C.
Today and tomorrow, Cure SMA’s Scientific Advisory Board (SAB) will be meeting in Washington D.C. Our Scientific Advisory Board is a recognized group of experts […]
Read More ›Families, Government, and Industry Leaders Unite to Help Cure SMA
This year’s 4th Annual Hope on the Hill Congressional Dinner in Washington D.C. is less than three weeks away. At Cure SMA, we know that […]
Read More ›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 […]
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