News

FightSMA and Gwendolyn Strong Foundation Announce Barrington G. Burnett As The $62,500 Emerging Investigator Award Winner

Dr. Barrington G. Burnett was named the recipient of the 2014 Emerging Investigator $62,500 Finalist Award, according to a recent Gwendolyn Strong Foundation and FightSMA announcement. Dr. Barrington G. Burnett’s project was entitled “Targeting the Ubiquitin Proteasome System to Treat Spinal Muscular Atrophy,” with the goal of identifying pathways and genes…

New Theory to Treat SMA Tested in the Laboratory

A new approach to treating spinal muscular atrophy (SMA) is in the works in the laboratory of Matthew E. R. Butchbach at the Nemours Alfred I. duPont Hospital for Children in Wilmington, Delaware. The team of researchers, which includes Drs. Sean G. Mack, Daniel J. Cook, and Prasad Dhurjati,…

AveXis Secures New Financing to Further SMA Clinical Studies

AveXis, Inc., a drug development company currently using gene therapy to address patients’ unmet needs suffering with orphan diseases such as Spinal Muscular Atrophy (SMA), has recently announced the completion of a $10 million financing set that was led by Deerfield Management and Roche Venture Fund. As part of the terms of…

Cytokinetics and Astellas Expand Development Collaboration Of Skeletal Muscle Activators To Treat Muscular Atrophy, Other Neuromuscular Indications

South San Francisco based Cytokinetics, Incorporated and Astellas Pharma Inc. of Tokyo, Japan, have announced amendments expanding their research collaboration alliance for development and commercialization of skeletal muscle activators. The two companies have been cooperating on research and development efforts, the objective being to advance novel treatment therapies…