We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This is the first of four total updates, and the first of two in basic research. This summary was written by Cure SMA Scientific Advisory Board member Adrian Krainer PhD, […]
2014 SMA Researcher Meeting Summary: The SMN Protein Read More »
We’re so excited to be the Groupon Coupon charity of the day for the #GrouponSaysThanks campaign! Shop at Groupon today and you’ll help support Cure SMA.
Cure SMA is Groupon’s Charity of the Day Read More »
Cure SMA recently announced the formation of the Subcommittee on Adult Outreach. This group will work to actively engage the adult SMA community in order to effectively support them. The population of adults with SMA is increasing, so this new subcommittee will play a vital role in shaping our programs in 2015 and beyond. Initially,
Cure SMA Announces New Subcommittee on Adults with SMA Read More »
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 type I, and a trial in children with SMA type II. Isis previously announced recruitment for a Phase III trial of infants with SMA type I. This is the first-ever
Isis Releases Phase II Clinical Trial Results Read More »
On Thursday, October 2, Jennifer Miller Smith and Fiorenna Fuentes Israel, two dedicated mothers of children with SMA, planned an evening to be remembered in their quest to achieve a huge fundraising milestone: $500,000 to cure spinal muscular atrophy. This year’s event, the 4th Annual Gala of Hope, has already raised $110,000 and climbing, bringing
South Florida Gala of Hope Closes in on Milestone Read More »
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 »
