New Avenue for Developing Treatments for Genetic Muscle-Wasting Disease

ScienceDaily (Mar. 16, 2010) — 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 research began as an attempt to understand the molecular pathways that are involved in SMA. Scientists have known for many years that this disease is caused by inherited mutations in a gene called survival motor neuron 1 (SMN1). These mutations cause nerve cells to lose their ability to control muscles, but researchers have never fully understood why.

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