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Spinal muscular atrophy

Spinal muscular atrophy (SMA) is the number one genetic cause of death in infants. Researchers at The Ottawa Hospital are leading cutting-edge studies to improve our understanding of SMA and develop better treatments. This page includes selected news stories about our SMA research, links to researchers working in this area and related resources. Learn more below.

Research News


Spinal muscular atrophy: Lab study shows importance of treating the liver to benefit a neuromuscular disorder Spinal muscular atrophy: Lab study shows importance of treating the liver to benefit a neuromuscular disorder
January 7, 2025 - “Our study suggests that treating the liver may be just as important as treating the neuromuscular system in patients with SMA. This is important because some of the approved SMA therapies are better at reaching the liver than others.”
Gene editing strategy shows promise in mice with spinal muscular atrophy Gene editing strategy shows promise in mice with spinal muscular atrophy
December 20, 2023 - Dr. Rashmi Kothary’s team played a key role in identifying a new gene-editing strategy that could someday be used to treat spinal muscular atrophy.
Whole-body approach needed to treat neuromuscular disease SMA Whole-body approach needed to treat neuromuscular disease SMA
July 13, 2022 - Treating a patient’s entire body, not just the nerves in their muscles, is essential for the complete treatment of spinal muscular atrophy (SMA), according to a study led by Aoife Reilly in Dr. Rashmi Kothary’s group.
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Related Resources

Neuromuscular disease research at The Ottawa Hospital

Search for clinical trials at The Ottawa Hospital related to this disease or condition.