Understanding and treating congenital myasthenic syndrome
Congenital myasthenic syndrome affects babies born with a defect inherited from their parents, which makes them unable to move and breathe. Some babies are more or less affected depending on the defect inherited from their parents and no cure exists for newborn with a severe form of the disease. Our group composed of a clinician and 2 scientists have identified the defect affecting two Albertan babies who were born at the Stollery Hospital with this disease. With this proposal, we will take our study to the next level with the aim to identify potential cures for babies born with the same defects and give them a chance to live.