Study opens new avenue for developing treatments for genetic muscle-wasting disease

Scientists from the Ottawa Hospital Research Institute (OHRI) and the University of Ottawa have identified a promising new approach for developing drugs to treat Spinal muscular atrophy (SMA), the leading inherited cause of death in infants and toddlers. Dr. Rashmi Kothary and his doctoral student Melissa Bowerman have found that an enzyme called RhoA is overly active in a mouse model of the disease and blocking this enzyme can greatly increase survival. The study is published in Human Molecular Genetics.

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