News

Gene Targeting Therapy Investigated in SMA Cells

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 Updates Clinical Development of RG7800 for SMA

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…

2015 SMA Researcher Meeting Summary on SMA Pathology

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…

Researchers Develop New Mice Models for SMA Types 2 and 3

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…

2015 SMA Researcher Meeting Summary on Drug Discovery

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…