Barry J Byrne MD PhD

Pediatric Cardiology•Gainesville, FL

Pediatric Cardiac Genetics, Pediatric Heart Failure and Heart Transplantation

Professor & Assoc. Chair of Pediatrics, University of Florida College of Medicine

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1200 Newell DriveARB, RG-120Gainesville, FL 32610

Education & Training

Johns Hopkins UniversityFellowship, Pediatric Cardiology, 1987 - 1990
Johns Hopkins UniversityResidency, Pediatrics, 1984 - 1987
University of Illinois College of MedicineClass of 1984

Certifications & Licensure

FL State Medical License 1997 - 2026
AL State Medical License 2020 - 2020
MD State Medical License 1985 - 2000
American Board of Pediatrics Pediatric Cardiology

Awards, Honors, & Recognition

Regional Top Doctor Castle Connolly, 2014
Fellow (FACC) American College of Cardiology

Clinical Trials

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease Start of enrollment: 2010 Sep 01
Completed
Phase 1, Phase 2
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) Start of enrollment: 2009 Jul 01
Completed
Phase 3
Extension Study for Patients Who Have Participated in a BMN 701 Study Start of enrollment: 2011 Aug 15
Terminated
Phase 2
Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies Start of enrollment: 2008 Oct 01
Completed
Newborn Screening for Critical Congenital Heart Disease Start of enrollment: 2010 Mar 01
Completed
A Study of Tadalafil for Duchenne Muscular Dystrophy Start of enrollment: 2013 Sep 01
Terminated
Phase 3
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) Start of enrollment: 2014 Apr 01
Terminated
Phase 3
A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction Start of enrollment: 2016 Oct 01
Terminated
Phase 1
Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy Start of enrollment: 2013 Dec 01
Completed
A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM) Start of enrollment: 2014 Sep 01
Completed
Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease Start of enrollment: 2017 Oct 17
Completed
Phase 1
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy Start of enrollment: 2015 Jun 30
Completed
Phase 2
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
COMPLETED
PHASE1, PHASE2
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) Start of enrollment: 2020 Jul 13
ACTIVE_NOT_RECRUITING
PHASE3
Roles: Principal Investigator
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Publications & Presentations

PubMed

1 citations
Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophy.
Barry J Byrne, Russell J Butterfield, Perry B Shieh, Edward C Smith, Christoph Licht
Molecular Therapy. 2025-09-03
1 citations
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy.
Jodi M Wolff, Nora Capocci, Evrim Atas, Diana X Bharucha-Goebel, John F Brandsema
Neuromuscular Disorders. 2025-08-23
1 citations
Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL.
Hani Kushlaf, Jordi Díaz-Manera, Drago Bratkovic, Barry J Byrne, Kristl G Claeys
Muscle & Nerve. 2025-08-01

Lectures

Barth Syndrome 6th International Scientific, Medical & Family Conference
Barth Syndrome Foundation, Saint Pete Beach, Florida - 6/25/2012

Press Mentions

Muscular Dystrophy Gene Therapy Nears Approval, but Safety Concerns LingerMay 23rd, 2023
Gene Therapy for Muscular Dystrophy Stirs Hopes and ControversyMay 2nd, 2023
4D Molecular Therapeutics Presents Interim Data from 4D-310 INGLAXA Phase 1/2 Clinical Trials & Development Plans for Fabry Disease Cardiomyopathy at WORLDSymposium™February 23rd, 2023
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Grant Support

Procession to IND of a capsid mutated AAV8 codon optimized NAGLU vector for treatment of Sanfilippo Syndrome type BUNIVERSITY OF FLORIDA2024–2026
Procession to IND of a capsid mutated AAV8 codon optimized NAGLU vector for treatment of Sanfilippo Syndrome type BUNIVERSITY OF FLORIDA2024–2026
Control of Breathing and Pompe DiseaseUNIVERSITY OF FLORIDA2007–2025
Vector CoreNational Heart, Lung, And Blood Institute2009–2011
Recombinant AAV For Correctional Genetic Abnormalities Project 4: Strategies ForNational Institute Of Diabetes And Digestive And Kidney Diseases2009
Phase I Trial Of Ocular Subretinal Injection Of A Raav2-Cb - Hrpe65National Center For Research Resources2008–2009
Cardiac And Skeletal Muscle In Barth SyndromeNational Center For Research Resources2008–2009
Aglu03206 OPEN Label Extension Of Aglu02704National Center For Research Resources2008–2009
Recombinant AAV For Correction Of Genetic AbnormalitiesNational Institute Of Diabetes And Digestive And Kidney Diseases2007–2009
Strategies For Sustained Effect Of Aav-Mediated Correction Of Pompe DiseaseNational Institute Of Diabetes And Digestive And Kidney Diseases2005–2009
Core--AdministrativeNational Institute Of Diabetes And Digestive And Kidney Diseases2007
Recombinant Human Acid Alpha-Glucosidase Trmt In PTS With Glycogen Storage DISNational Center For Research Resources2004–2007
(RHGAA) Trmt In PTS 6 Month W/Infantile-Onset Pompe DiseaseNational Center For Research Resources2006
Ngvl Toxicology LaboratoryNational Center For Research Resources2005–2006
(RHGAA) Trmt In PTS U 6 Monti W/Infantile-Onset Pompe DiseaseNational Center For Research Resources2005
Correction Of Inherited Cardiomyopathy Using AAV VectorsNational Heart, Lung, And Blood Institute1998–2005
Glycogen Storage Disease Treatment: Hepatic Gene TherapyNational Institute Of Diabetes And Digestive And Kidney Diseases2000–2002
Gene Therapy For Glycogen Storage DiseaseNational Institute Of Diabetes And Digestive And Kidney Diseases2000–2002
Correction Of Inherited Cardiomyopathy Using AAV VectorsNational Heart, Lung, And Blood Institute1997–2001