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

Education Matters

Parent Project msucular Dystrophy

Duchenne Resources for Back-To-School

As the beginning of the school year quickly approaches, PPMD is working to support your family and help you plan for a successful classroom experience, whether it be in-person or virtual.


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



Bright star in fight against rare disease

Dr. Barry Byrne has a unique ability to inspire others as he fights for his patients’ lives



Breathing free again

UF performs first diaphragm pacing surgery on a patient with Pompe disease

Winter 2011


The promise of a new home

The Fox family not only found a new home in Gainesville, they found new hope.

Fall 2009

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

Explore More

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.

Dr. Barry Byrne and Phoenix


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.

Disabled Child

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…

Category: News

PepGen raises $113M to challenge Sarepta in DMD

Category: Duchenne Muscular Dystrophy

Sarepta Acquires Investigational Gene Therapy for…

Category: Limb-Girdle Muscular Dystrophy