Breakthroughs happen here…
At the Powell Center for Rare Disease Research we perform research in a vast range of specialties, revolutionizing treatments for children with rare and complex disorders.
Friedreich’s ataxia Research
FARA Global Challenge
Tour Our Friedreich’s Ataxia Research Facilities
Meet Dr. Byrne and the team at the University of Florida! In this video, you will meet Dr. Byrne who will introduce you to their hardworking and dynamic team at the University of Florida. Do you spot anyone familiar? #rideATAXIAGlobalChallenge
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UF Innovator of the Year
2022 Innovator of the Year
Dr. Barry Byrne Named Innovator of the Year (UF Innovate)
“If Dr. Byrne were here, he’d take the stage, accept this large lighted trophy, and likely inspire you to keep working with UF Innovate | Tech Licensing as researchers – and consider entrepreneurship, too,” said Dr. David Norton, vice president for UF Research.
Clinical Research Center
University of Florida College of Medicine
Clinical Trials
A clinical trial is a research study that looks into the safety and usefulness of a drug in improving symptoms or slowing, stopping, or reversing the progression of a disease. Most drugs tested in clinical trials are not yet available to the public (Phase I, II, III studies) while some studies involve medications that are already available (Phase IV studies).
FLORIDA PHYSICIAN FEATURES
DOUG BENNETT, BILL LEVESQUE
Written in our DNA
UF researchers’ groundbreaking work from 1980s leads to new treatment for debilitating neuromuscular disease
MEGAN D. KIMMEL
Bright star in fight against rare disease
Dr. Barry Byrne has a unique ability to inspire others as he fights for his patients’ lives
MELANIE STAWICKI AZAM
Breathing free again
UF performs first diaphragm pacing surgery on a patient with Pompe disease
PRISCILLA SANTOS
The promise of a new home
The Fox family not only found a new home in Gainesville, they found new hope.
Pediatric Neuromuscular Clinic
UF Health Shands Hospital
Center for Pediatric Neuromuscular and Rare Diseases
The pediatric neuromuscular program at UF Health Shands Hospital offers one-stop access to comprehensive care, including leading-edge diagnostic techniques, medical treatments and research. Our neuromuscular program cares for infants, children and teens with neuromuscular disorders.
Our Director
Barry J. Byrne, MD, PhD
Dr. Barry Byrne is the Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center. He obtained his B.S. degree from Denison University, his M.D. and Ph.D. from the University of Illinois. He completed his Pediatrics residency, cardiology fellowship training and post-doctoral training in Biological Chemistry at the Johns Hopkins Hospital. He joined the University of Florida in 1997 and is now the Earl and Christy Powell University Chair in Genetics.
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Powell Center for Rare Disease research
Clinical Research
Our research team conduct various clinical trials, working to identify improved therapies. As a result of this research, our patients are among the first to benefit from new discoveries.
CENTER FOR PEDIATRIC NEUROMUSCULAR AND RARE DISEASES
Neuromuscular Program
Management of neuromuscular disorders requires multidisciplinary expertise. The pediatric neuromuscular program at UF Health Shands Hospital offers one-stop access to comprehensive care, including leading-edge diagnostic techniques, medical treatments and research.
Coming Soon…
Neuromuscular Rehabilitation
Consistent and frequent neuromuscular rehabilitation is one of the few treatments, at this time, for all with a neuromuscular disease. It’s important through all stages and ages to potentially delay progression of the disease.
Industry Partners
UF Innovate
Gene Therapy Company AavantiBio Launches With $107 Million Series A Financing
Headquartered in the greater Boston area, AavantiBio is co-founded by renowned gene therapy researchers Barry Byrne, M.D., Ph.D., and Manuela Corti, P.T., Ph.D., who together bring thirty years of experience to the Company.
Sarepta Therapeutics
Sarepta Therapeutics and University of Florida Announce Collaboration
Sarepta Therapeutics Inc., the leader in precision genetic medicine for rare diseases, and the University of Florida today announced a strategic collaboration to enable cutting-edge research for novel genetic medicines. Through the agreement, Sarepta will fund multiple research programs at the University, and will have an exclusive option to further develop any new therapeutic compounds that result from the funded research programs.
In the news
Zolgensma-Evrysdi Combo Likely Beneficial for SMA…
PepGen raises $113M to challenge Sarepta in DMD
Sarepta Acquires Investigational Gene Therapy for…
