Experimental therapy halts progress of Toronto boy’s neurodegenerative disease in rare breakthrough

A gene therapy made specially for a Toronto boy with an ultrarare genetic disorder is safe and appears to have halted the progression of his disease, according to a new paper outlining the results of a single-patient clinical trial that his family raised millions of dollars to fund.When Michael Pirovolakis, now 6, was diagnosed with spastic paraplegia type 50 (SPG50) at 16 months of age, he was the only patient in Canada known to have the disorder.