BarryJByrneMDPhD

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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Office
1200 Newell Drive
ARB, RG-120
Gainesville, FL 32610
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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 Start of enrollment: 2016 Jan 01
Active, not recruiting
Phase 1, Phase 2
A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) Start of enrollment: 2020 Jul 13
Active, not recruiting
Phase 3
Roles: Principal Investigator
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Publications & Presentations

PubMed

2 citations
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
Barry J Byrne, Benedikt Schoser, Priya S Kishnani, Drago Bratkovic, Paula R Clemens, Ozlem Goker-Alpan, Xue Ming, Mark Roberts, Matthias Vorgerd, Kumaraswamy Sivakumar...> ;Journal of Neurology. 2024 Apr 1
7 citations
Meeting Report: 2022 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Transfer Therapy'.
Angela Lek, Evrim Atas, Sharon E Hesterlee, Barry J Byrne, Carsten G Bönnemann> ;Journal of Neuromuscular Diseases. 2023 Jan 1
Nicholas Muzyczka, PhD.
Kenneth I Berns, Barry J Byrne, Terence R Flotte, R Jude Samulski, Arun Srivastava> ;Molecular Therapy. 2023 Mar 1
Nicholas Muzyczka, PhD [1947-2023].
Kenneth I Berns, Barry J Byrne, Terence R Flotte, R Jude Samulski, Arun Srivastava> ;Human Gene Therapy. 2023 Feb 1
15 citations
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy.
Martin Schulz, Daniel I Levy, Christos J Petropoulos, George Bashirians, Ian Winburn, Matthias Mahn, Suryanarayan Somanathan, Seng H Cheng, Barry J Byrne> ;Molecular Therapy. 2023 Mar 1
15 citations
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
Sharla M Birch, Michael W Lawlor, Thomas J Conlon, Lee-Jae Guo, Julie M Crudele, Eleanor C Hawkins, Peter P Nghiem, Mihye Ahn, Hui Meng, Margaret J Beatka, Brittany A ...> ;Science Translational Medicine. 2023 Jan 4
5 citations
Secondary hemophagocytic lymphohistiocytosis following Zolgensma therapy: An evolving story on the innate response to systemic gene therapy.
Barry J Byrne, Melissa Elder, Carmen Leon-Astudillo, Manuela Corti> ;Molecular Therapy. 2022 Dec 7
7 citations
Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec.
Carmen Leon-Astudillo, Mary Wagner, Stephanie M Salabarria, Jenna Lammers, Julie Berthy, Carla D Zingariello, Barry J Byrne, Barbara K Smith> ;Sleep Medicine. 2023 Jan 1
2 citations
Chemogenetic activation of hypoglossal motoneurons in a mouse model of Pompe disease.
Michele L Singer, Sabhya Rana, Ethan S Benevides, Brian E Barral, Barry J Byrne, David D Fuller> ;Journal of Neurophysiology. 2022 Nov 1
4 citations
Longitudinal Observational Study of Cardiac Outcome Risk Factor Prediction in Children, Adolescents, and Adults with Barth Syndrome.
Shahryar Chowdhury, Lanier Jackson, Barry J Byrne, Randall M Bryant, W Todd Cade, Tammy Lane Churchill, Julia Buchanan, Carolyn Taylor> ;Pediatric Cardiology. 2022 Aug 1
3 citations
Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging.
Abhinandan Batra, Alison M Barnard, Donovan J Lott, Rebecca J Willcocks, Sean C Forbes, Saptarshi Chakraborty, Michael J Daniels, Jannik Arbogast, William Triplett, Er...> ;BMC Cardiovascular Disorders. 2022 Jun 9
9 citations
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
Mazen M Dimachkie, Richard J Barohn, Barry Byrne, Ozlem Goker-Alpan, Priya S Kishnani, Shafeeq Ladha, Pascal Laforêt, Karl Eugen Mengel, Loren D M Peña, Sabrina Saccon...> ;Neurology. 2022 Aug 1
11 citations
Comparison of highly pure rAAV9 vector stocks produced in suspension by PEI transfection or HSV infection reveals striking quantitative and qualitative differences.
Prasad D Trivedi, Chenghui Yu, Payel Chaudhuri, Evan J Johnson, Tina Caton, Laura Adamson, Barry J Byrne, Nicole K Paulk, Nathalie Clément> ;Molecular Therapy. Methods & Clinical Development. 2022 Mar 10
1 citations
Advanced therapeutic strategy for hereditary neuromuscular diseases.
Wuh-Liang Hwu, Shin-Ichi Muramatsu, Yin-Hsiu Chien, Barry J Byrne> ;Molecular Therapy. 2022 Jan 5
Addressing the implementation gap in advanced therapeutics for spinal muscular atrophy in the era of newborn screening programs.
Carmen Leon-Astudillo, Barry J Byrne, Ramzi G Salloum> ;Frontiers in Neurology. 2022 Jan 1
30 citations
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, d...
Benedikt Schoser, Mark Roberts, Barry J. Byrne, Sheela Sitaraman, Hai Jiang, Pascal Laforet, Antonio Toscano, Jeff Castelli, Jordi Díaz-Manera, Mitchell Goldman, Ans T...> ;The Lancet. Neurology. 2021 Dec 1
45 citations
Long-term efficacy and safety of eladocageneexuparvovec in patients with AADC deficiency
Chun-Hwei Tai, Ni-Chung Lee, Yin-Hsiu Chien, Barry J. Byrne, Shin-ichi Muramatsu, Sheng-Hong Tseng, Wuh-Liang Hwu> ;Molecular Therapy. 2021 Nov 8
3 citations
Scratching the surface of RGD-directed AAV capsid engineering.
Sergei Zolotukhin, Prasad D. Trivedi, Manuela Corti, Barry J. Byrne> ;Molecular Therapy. 2021 Oct 25
5 citations
Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort.
Barry J. Byrne, Steven D. Colan, Priya S. Kishnani, Meredith C. Foster, Susan Sparks, James B. Gibson, Kristina An Haack, David W. Stockton, Loren D.M. Pena, Si Houn H...> ;Cardiology in the Young. 2021 Aug 23
23 citations
A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirse...
Olga Mitelman, Hoda Z. Abdel-Hamid, Barry J. Byrne, Anne M. Connolly, Peter Heydemann, Crystal Proud, Perry B. Shieh, Kathryn R. Wagner, Ashish Dugar, Sourav Santra, J...> ;Journal of Neuromuscular Diseases. 2021 Aug 19
1 citations
Resistance exercise training with protein supplementation improves skeletal muscle strength and improves quality of life in late adolescents and young adults with Bart...
Kathryn L. Bohnert, Grace Ditzenberger, Adam J. Bittel, Lisa de las Fuentes, Manuela Corti, Christina A. Pacak, Carolyn L. Taylor, Barry J. Byrne, Dominic N. Reeds, W....> ;JIMD Reports. 2021 Aug 9
4 citations
Case Studies in Neuroscience: Neuropathology and diaphragm dysfunction in ventilatory failure from late-onset Pompe disease.
David D. Fuller, Jorge A. Trejo-Lopez, Anthony T. Yachnis, Michael D. Sunshine, Sabhya Rana, Victoria E. Bindi, Barry J. Byrne, Barbara K. Smith> ;Journal of Neurophysiology. 2021 Jun 30
6 citations
Phenotypic implications of pathogenic variant types in Pompe disease.
Manuel Viamonte, Stephanie L. Filipp, Zara Zaidi, Matthew J. Gurka, Barry J. Byrne, Peter B. Kang> ;Journal of Human Genetics. 2021 May 11
Mavis Agbandje-McKenna's lifelong commitment to teaching and research.
Barry J. Byrne, Angela L McCall, Beverly L. Davidson, R. Jude Samulski> ;Molecular Therapy. 2021 Apr 24
13 citations
Clinical, neuroimaging and molecular spectrum of TECPR2-associated hereditary sensory and autonomic neuropathy with intellectual disability.
Sonja Neuser, Barbara Brechmann, Gali Heimer, Ines Brösse, Susanna Schubert, Lauren O’Grady, Michael Zech, Siddharth Srivastava, David A. Sweetser, Yasemin Dincer, Vol...> ;Human Mutation. 2021 Apr 13
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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

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