Leroy, Bart P.

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Russell, S., Bennett, J., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., McCague, S., et al. (2017) · The Lancet

Retinal Development and DisordersVirus-based gene therapy research

Cited by 1,952

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Maguire, A. M., High, K. A., Auricchio, A., Wright, J. F., Pierce, E. A., Testa, F., Mingozzi, F., Bennicelli, J. L., Ying, G.-S., Rossi, S., et al. (2009) · The Lancet

Retinal Development and DisordersVirus-based gene therapy research

Cited by 865

Retinal layer segmentation in multiple sclerosis: a systematic review and meta-analysis

Petzold, A., Balcer, L. J., Calabresi, P. A., Costello, F., Frohman, T. C., Frohman, E. M., Martinez-Lapiscina, E. H., Green, A. J., Kardon, R., Outteryck, O., et al. (2017) · The Lancet Neurology

Multiple Sclerosis Research StudiesGlaucoma and retinal disorders

Cited by 585

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Bennett, J., Wellman, J., Marshall, K. A., McCague, S., Ashtari, M., DiStefano-Pappas, J., Elci, O. U., Chung, D. C., Sun, J., Wright, J. F., et al. (2016) · The Lancet

Retinal Development and DisordersVirus-based gene therapy research

Cited by 461

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy

Maguire, A. M., Russell, S., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., Marshall, K. A., et al. (2019) · Ophthalmology

Retinal Development and DisordersVirus-based gene therapy research

Cited by 409

Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease

Maguire, A. M., Russell, S., Chung, D. C., Yu, Z.-F., Tillman, A., Drack, A. V., Simonelli, F., Leroy, B. P., Reape, K. Z., High, K. A., et al. (2021) · Ophthalmology

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 194

Real-world safety and effectiveness of voretigene neparvovec: results up to 2 Years from the prospective, registry-based PERCEIVE study

Fischer, M. D., Simonelli, F., Sahni, J., Holz, F. G., Maier, R., Fasser, C., Suhner, A., Stiehl, D. P., Chen, B., Audo, I., et al. (2024) · Biomolecules

Retinal Development and DisordersVirus-based gene therapy research

Cited by 70

Gene therapy for inherited retinal disease: Long-term durability of effect

Leroy, B. P., Fischer, M. D., Flannery, J. G., MacLaren, R. E., Dalkara, D., Scholl, H. P. N., Chung, D. C., Spera, C., Viriato, D., & Banhazi, J. (2023) · Ophthalmic Research

Retinal Development and DisordersVirus-based gene therapy research

Cited by 70

Adult Refsum disease: A form of tapetoretinal dystrophy accessible to therapy.

Rüether, K., Baldwin, E., Casteels, M., Feher, M. D., Horn, M., Kuranoff, S., Leroy, B. P., Wanders, R. J., & Wierzbicki, A. S. (2010) · Survey of Ophthalmology

Metabolism and Genetic DisordersRetinal Development and Disorders

Cited by 42

Syndromic retinitis pigmentosa.

Karuntu, J. S., Almushattat, H., Nguyen, X.-T.-A., Plomp, A. S., Wanders, R. J. A., Hoyng, C. B., van Schooneveld, M. J., Schalij-Delfos, N. E., Brands, M. M., Leroy, B. P., et al. (2025) · Progress in Retinal and Eye Research

Retinal Development and DisordersGenetic and Kidney Cyst Diseases

Cited by 39

Unilateral cancer-associated retinopathy: diagnosis, serology and treatment

Roels, D., Ueno, S., Talianu, C. D., Draganova, D., Kondo, M., & Leroy, B. P. (2017) · Documenta Ophthalmologica

Autoimmune Neurological Disorders and TreatmentsOcular Diseases and Behçet’s Syndrome

Cited by 35

Safety of lenadogene nolparvovec gene therapy over 5 Years in 189 patients with leber hereditary optic neuropathy

VIGNAL-CLERMONT, C., YU-WAI-MAN, P., NEWMAN, N. J., CARELLI, V., MOSTER, M. L., BIOUSSE, V., SUBRAMANIAN, P. S., WANG, A.-G., DONAHUE, S. P., LEROY, B. P., et al. (2023) · American Journal of Ophthalmology

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 25