Isis Pharmaceuticals announced that it will initiate phase 3 clinical trials to study their investigational treatment for spinal muscular atrophy (SMA), ISIS-SMNRx. The phase 3 study, dubbed CHERISH, will include about 120 non-ambulatory children suffering from the severe and rare genetic neuromuscular disease, and is already the…
Dallas, Texas-based AveXis, Inc., a biotechnology company working to improve the quality of life of patients suffering from severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA) through the use of gene therapy, is launching a new website specifically dedicated to SMA, called SMAStudy.org. Focused on…
John Carbona, CEO of AveXis, along with Dr. Brian Kaspar have been invited to present the company’s latest developments at the 26th Annual Piper Jaffray Healthcare Conference, which will take place on Tuesday, December 2. AveXis is a biopharmaceutical company currently developing gene therapy-based treatments for severe genetic and orphan diseases such as spinal muscular atrophy (SMA) in order…
This year, Cure SMA granted the 2014 SMA Breakthrough Award to Isis Pharmaceuticals at the Cure SMA Gala of Hope, which took place in Seal Beach, California. The award was granted to the company in recognition of their work and efforts to improve treatment of spinal muscular atrophy (SMA), the leading genetic cause of…
Next Saturday, November, 8, Santa Barbara will host dozens of racers and walkers to raise both awareness and funds to support Spinal Muscular Atrophy (SMA) patients, at the annual Santa Barbara Veteran’s Day Marathon and Half Marathon. Gwendolyn Strong, a 7-year-old patient and the beneficiary of the event, dubbed the Gwendolyn…
Isis Pharmaceuticals received a $5 million milestone payment from partner GlaxoSmithKline following news of its investigational drug ISIS-GSK5Rx being granted the development candidate designation. ISIS-GSK5Rx is the second medication created as a result of the collaboration between the two companies and is an antisense drug, indicated for the treatment of an undisclosed ocular disease. Among other…
The organization “Families of SMA” which has been working since 1984 to help families suffering the burden of have a child with spinal muscular atrophy, recently announced that it has rebranded itself as “Cure SMA,” refocusing its work on finding a cure for the disease and supporting cutting-edge SMA research. The…
Get regular updates to your inbox.