2014 National Goodwill Ambassador announcement at Harley-Davidson headquarters, in Milwaukee Nine-year-old Reagan Imhoff has been chosen as the 2015 National Goodwill Ambassador, after spending 2014 traveling across the country to give a face and a voice to the Muscular Dystrophy Association (MDA). Despite the difficulties of suffering from…
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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 that…
Spinal muscular atrophy (SMA) is a genetic condition that is typically first diagnosed in infants and young children and is associated with high mortality in this population. SMA causes muscle weakness and progressive loss of movement. SMA is triggered by progressive deterioration in the nerve cells connecting the brain…
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, 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…
A recent review entitled “Neurodegeneration in spinal muscular atrophy: from disease phenotype and animal models to therapeutic strategies and beyond,” recently published the journal Future Neurology, discussed current evidence on SMA with a specific focus on the existing treatment options. Spinal muscular atrophy (SMA) is an autosomal…
A study entitled “The effect of diet on the protective action of D156844 observed in spinal muscular atrophy mice” examined the effect of maternal diet in the gene SMN2 inducer in mice models with spinal muscular atrophy. The study was published in the journal Experimental Neurology. Proximal…
A recent review of current evidence on the pathogenesis of SMA based on the gene SMN was conducted by Kathryn Swoboda from the Department of Neurology, University of Utah School of Medicine. The perspectives Dr. Swoboda offers in the review suggest that experimental SMN-targeted therapeutics are indeed highly promising for treating…
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…
Spinal Muscular Atrophy (SMA) is a genetic disorder that causes progressive muscle wasting and robs the afflicted of physical strength and leads to mobility impairment due to its effect on motor nerve cells in the spinal cord. SMA eventually results in loss of the ability to walk, eat,…
