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Dr. Barry Byrne has a way with people. While he has a
stellar reputation as a world-class physician-scientist who is
pioneering the use of gene therapy for debilitating and rare
diseases, it is his charismatic and energetic personality that
draws people to him for their care or their careers, or to help
advance research in rare diseases.
Byrne, who earned his medical degree
and PhD from the University of Illinois and
completed postdoctoral training and residency
at The Johns Hopkins University, arrived at
the UF College of Medicine in 1997. He is
associate chair of research in the department
of pediatrics, the Earl and Christy Powell
University Chair in Gene Therapy and Genetics
Research, director of the UF Powell Center for
Rare Disease Research and Therapy, a professor,
father, friend and a warrior in the fight against
genetic muscular diseases. He leads the fight
by developing treatments to fix the gene
abnormalities, deletions and errors that wreak
havoc in people’s bodies.
“A rare disease diagnosis significantly impacts
patients and their families because many times
there is no cure. It’s a challenge that requires the
whole family to adapt and learn what it means
to live with these diseases,” Byrne said. “Our
team focuses on the patient and looks for ways
we can improve their life, or maybe find a cure
through our research.”
Byrne is one of the world’s leading experts
on Pompe disease, a rare inherited genetic
disorder that prevents muscles from functioning
properly, especially the lungs and heart. He and
his multidisciplinary team study and look for
promising treatment and therapies in several
other genetic diseases such as Duchenne
muscular dystrophy, Barth syndrome, Canavan
disease and others that affect muscles.
2018 “A RARE DISEASE DIAGNOSIS
—Dr. Barry Byrne
From front to back:
Barry Byrne, MD, PhD; Tina
Cousins, RDCS; Brian Cleaver, PhD;
Samantha May, Nikki Atzinger,
Jessica Bowman and Gee Kim.