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

Global Challenge Play Video

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.

Innovator fo the Year

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).

Boy wearing a blue hat sitting in a wheel chair holding stuffed shark



Written in our DNA

UF researchers’ groundbreaking work from 1980s leads to new treatment for debilitating neuromuscular disease


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.

Little boy with freckles hugging a dog

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.

Barry Byrne

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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.

Duchenne Awareness Month


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.

Research Subject

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.

Research Subject

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.