Dallas-based gene therapy platform company AveXis Inc., a leader in developing treatments for rare and life-threatening neurological genetic diseases, has announced completion of a $65 million Class D common stock financing. The proceeds will be used to advance the company’s ongoing Spinal Muscular Atrophy (SMA) research and clinical programs…
When a protein is missing in a cell, as in the case of spinal muscular atrophy (SMA) where survival motor neuron (SMN) is missing, a variety of methods can be used to restore function to cells lacking the protein and treat the disease. Direct delivery of the protein or a…
Genentech, a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions, recently announced an update on the clinical development of RG7800, its investigational SMN2 splicing modifier that is being developed for the treatment of patients with spinal muscular atrophy…
The 19th International SMA Researcher Meeting was recently held by Cure SMA in Kansas City, Missouri (June 18-20, 2015). SMA (spinal muscular atrophy) is a rare, devastating motor neuron disease and one of the leading genetic causes of pediatric mortality, occurring in approximately 1 in every 6,000 to 10,000 newborns. It…
A study recently published in the journal Molecular Genetics and Genomic Medicine reported a new assay to assess the copy number of specific genes linked to spinal muscular atrophy (SMA) pathogenesis and disease severity. The study was led by researchers at the Nemours/Alfred I. duPont Hospital…
Researchers at the University College London in the United Kingdom recently reported the development of new mice models with intermediate clinical severity of spinal muscular atrophy (SMA). The study, published in the journal Human Molecular Genetics, is titled “Repeated low doses of morpholino antisense…
Researchers have understood the genetic basis of spinal muscular atrophy (SMA) for many years. Mutations in the gene that encodes the protein survival motor neuron (SMN1) are the most usual cause of disease, as a mutation leads to a deficiency of SMN. Without this protein, motor neurons die, leading to…
Researchers at Rockefeller University and Columbia University in New York recently reported a new technique which enabled them to identify specific molecular determinants linked to Parkinson’s disease and other neurodegenerative disorders, including spinal muscular atrophy (SMA). The study was published in the journal Nature Neuroscience and…
The 19th International SMA Researcher Meeting has been recently held by Cure SMA at Kansas City, Missouri (June 18-20, 2015). Summaries with the most relevant developments and findings presented at the meeting are currently being made available. SMA (spinal muscular atrophy) is a rare, devastating motor neuron disease…
The 19th International SMA Researcher Meeting was recently held by Cure SMA in Kansas City, Missouri (June 18-20, 2015). Cure SMA announced that summaries from the 2015 meeting will be available showing the most relevant developments and findings presented at the meeting. SMA (spinal muscular atrophy) is a rare, devastating motor…