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Office
1200 Newell Drive
ARB, RG-120
Gainesville, 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
- Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) Start of enrollment: 2009 Jul 01
- Extension Study for Patients Who Have Participated in a BMN 701 Study Start of enrollment: 2011 Aug 15
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Publications & Presentations
PubMed
- 485 citationsGene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 yearsSamuel G. Jacobson, Artur V. Cideciyan, R. Ratnakaram, Elise Héon, Sharon B. Schwartz
Archives of Ophthalmology. 2012-01-09 - 719 citationsRecruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy.Jill Miller, Carl R. Urbinati, Patana Teng-umnuay, Myrna G. Stenberg, Barry J. Byrne
The EMBO Journal. 2000-09-01 - 9 citationsMicro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle DiseaseChady H. Hakim, Nathalie Clement, Lakmini P. Wasala, Hsiao T. Yang, Yongping Yue
Molecular Therapy. Methods & Clinical Development. 2020-07-09
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
Professional Memberships
- Member
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