First Patient Receives Gene Therapy for GSD 1a

First Patient Receives Gene Therapy for GSD 1a

UCONN Today Dr. David Weinstein and his team at UConn Health have administered to a patient the world’s first investigational gene therapy for potentially deadly glycogen storage disease (GSD). On July 24, the novel gene therapy to treat the rare childhood genetic...

Pompe Gene Therapy Found Safe During Human Trial

From UF Health UfHealth.org A gene therapy that treats respiratory problems in early-onset Pompe disease was shown to be safe during its first human trial, University of Florida Health researchers have found. The inherited disease causes a complex sugar to accumulate...

Hope to Go Home Tomorrow

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