How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years

<p>Michael Pirovolakis, a four-year-old with the ultra-rare, neurodevelopmental condition SPG50 disease, earlier this year became the first person to be dosed with an experimental gene therapy developed to treat the disorder. The gene therapy was the result of a relentless pursuit by his parents, Terry and Georgia, to raise money and engage scientists and others in the development of a treatment for SPG50. We spoke to Michael’s father and founder of CureSPG50 Terry Pirovolakis and associate professor at UT Southwestern Medical Center Steven Gray, about SPG50, the work to develop and advance an experimental gene therapy for the condition into the clinic, and why Pirovolakis says his work is not yet done.&nbsp;</p>