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 infants with spinal muscular atrophy (SMA). The trial (NCT02122952) opened for enrollment in April 2014. “History in gene transfer therapy was made on May 13th, […]
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 of the 19th International World Muscle Society Congress in Berlin, Germany. The presentation is also available via a live streaming webcast and conference call. When:
Isis Presents Phase II Trial Results Read More »
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 of drugs for conditions like SMA. In the chariSMA program, researchers are using the AAV9 virus to “infect” a cell with new DNA from survival
AveXis Receives Orphan Drug Designation Read More »
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 assess the clinical efficacy and safety of ISIS-SMNRx administered intrathecally (through an injection in the lower back, similar to an epidural) in patients with SMA.
Isis Begins Recruiting for Phase III Trial Read More »
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 flight of golfers, supporters, and friends enjoying a day of SMA awareness on the greens, followed by an incredible evening of cocktails, dinner, dancing, and
Cole P Randall Memorial Golf Tournament Raises $75,000 Read More »
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 of the University of Southern California. At Cure SMA, we know that no single group can develop a treatment for SMA alone—it requires collaboration. One
Cure SMA-Funded CALIBR Research Collaboration Expanded to USC Read More »
On Thursday, September 18, executives from the Barney & Barney Foundation presented Cure SMA with a check for $20,000 to support spinal muscular atrophy research and family support. Barney & Barney was started in San Diego in 1909 by two brothers. Since then, they have become one of the largest brokerages in the United States.
Cure SMA Receives Generous Donation from Barney & Barney Read More »
Over the last decade, we’ve seen many advances in spinal muscular atrophy research, from new techniques in gene therapy to drugs that show promise in slowing or stopping the progress of the disease. With such great promise in the research landscape, we know that no single group can develop a treatment for SMA alone—it requires
Catch Up on Research News from our Family Friendly Poster Session Read More »
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 contest is open to children with spinal muscular atrophy and their siblings. We will be posting a gallery of all entries on our Facebook page. We’ll also be selecting several
Cure SMA and Isis Announce Holiday Card Contest TEST Read More »
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 contest is open to children with spinal muscular atrophy and their siblings. We will be posting a gallery of all entries on our Facebook page. We’ll also be selecting several
Cure SMA and Isis Announce Holiday Card Contest Read More »
