How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years
RARECast - A podcast by RARECast - Thursdays
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Founder of CureSPG50 Terry Pirovolakis and associate professor at UT Southwestern Medical Center Steven Gray discuss ultra-rare neurodevelopmental condition 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.