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Funding for research into Spinal muscular atrophy

April 14, 2014

Dr. Rashmi Kothary has been awarded $253,800 over three years from the Muscular Dystrophy Association to further his research into spinal muscular atrophy, a disease affecting neurons and skeletal muscle that impacts very young children, leading to paralysis for which there is currently no effective treatment. Spinal muscular atrophy is the most common genetic disease resulting in infant death and is caused by mutations in a single gene, SMN1. Dr. Kothary and his team are developing a targeted gene therapy to fix the biological pathways disrupted by the disease. Efforts at gene-modulation therapy are close to the clinical trial stage, leading to the most optimism this field of research has seen in years.

About the Ottawa Hospital Research Institute
The Ottawa Hospital Research Institute (OHRI) is the research arm of The Ottawa Hospital and is an affiliated institute of the University of Ottawa, closely associated with the university’s Faculties of Medicine and Health Sciences. OHRI includes more than 1,700 scientists, clinical investigators, graduate students, postdoctoral fellows and staff conducting research to improve the understanding, prevention, diagnosis and treatment of human disease.

Media contact
Paddy Moore
Communications and Public Relations
Ottawa Hospital Research Institute
613-737-8899 x73687
613-323-5680 (cell)
padmoore@ohri.ca