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Lead investigator

Collaborators & Co-Investigators

• Emma Sutton, PhD

Research Sites & Affiliations

• Ottawa Hospital Research Institute, Ottawa, Ontario

Budget: $100,000

Disorders: Spinal muscular atrophy (5q-SMN)

Research Areas: Discover Novel Treatments & Therapies

Abstract: 

This study aims to discover a new way to treat spinal muscular atrophy (SMA) in babies before they are born. At the moment, treatment is given to babies after they are born, but this study aims to determine whether the treatment works better if given to the baby while the mother is still pregnant. This study will use mice with SMA to see if giving the treatment to the mother can stop the disease from causing problems in the baby. The results of this study have the potential to If this research works, it could mean that treating the mother with before the baby is born might be a new and better way to treat SMA patients.

Impact: