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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1993 1
2003 2
2004 2
2005 1
2006 4
2007 5
2008 5
2009 6
2010 8
2011 12
2012 9
2013 6
2014 6
2015 14
2016 15
2017 12
2018 13
2019 15
2020 11
2021 22
2022 14
2023 9
2024 3

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159 results

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Page 1
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 JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM. Russell S, et al. Among authors: leroy bp. Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14. Lancet. 2017. PMID: 28712537 Free PMC article. Clinical Trial.
Adult Refsum Disease.
Waterham HR, Wanders RJA, Leroy BP. Waterham HR, et al. Among authors: leroy bp. 2006 Mar 20 [updated 2021 Sep 30]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. 2006 Mar 20 [updated 2021 Sep 30]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. PMID: 20301527 Free Books & Documents. Review.
Cost-effective sequence analysis of 113 genes in 1,192 probands with retinitis pigmentosa and Leber congenital amaurosis.
Panneman DM, Hitti-Malin RJ, Holtes LK, de Bruijn SE, Reurink J, Boonen EGM, Khan MI, Ali M, Andréasson S, De Baere E, Banfi S, Bauwens M, Ben-Yosef T, Bocquet B, De Bruyne M, de la Cerda B, Coppieters F, Farinelli P, Guignard T, Inglehearn CF, Karali M, Kjellström U, Koenekoop R, de Koning B, Leroy BP, McKibbin M, Meunier I, Nikopoulos K, Nishiguchi KM, Poulter JA, Rivolta C, Rodríguez de la Rúa E, Saunders P, Simonelli F, Tatour Y, Testa F, Thiadens AAHJ, Toomes C, Tracewska AM, Tran HV, Ushida H, Vaclavik V, Verhoeven VJM, van de Vorst M, Gilissen C, Hoischen A, Cremers FPM, Roosing S. Panneman DM, et al. Among authors: leroy bp. Front Cell Dev Biol. 2023 Feb 3;11:1112270. doi: 10.3389/fcell.2023.1112270. eCollection 2023. Front Cell Dev Biol. 2023. PMID: 36819107 Free PMC article.
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.
Maguire AM, Russell S, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, Marshall KA, McCague S, Reichert H, Davis M, Simonelli F, Leroy BP, Wright JF, High KA, Bennett J. Maguire AM, et al. Among authors: leroy bp. Ophthalmology. 2019 Sep;126(9):1273-1285. doi: 10.1016/j.ophtha.2019.06.017. Epub 2019 Jun 22. Ophthalmology. 2019. PMID: 31443789 Free article. Clinical Trial.
RNA-based therapies in inherited retinal diseases.
Girach A, Audo I, Birch DG, Huckfeldt RM, Lam BL, Leroy BP, Michaelides M, Russell SR, Sallum JMF, Stingl K, Tsang SH, Yang P. Girach A, et al. Among authors: leroy bp. Ther Adv Ophthalmol. 2022 Nov 4;14:25158414221134602. doi: 10.1177/25158414221134602. eCollection 2022 Jan-Dec. Ther Adv Ophthalmol. 2022. PMID: 36388727 Free PMC article. Review.
Hydroxychloroquine hitting the headlines-retinal considerations.
Downes SM, Leroy BP, Sharma SM, Sivaprasad S, Dollfus H. Downes SM, et al. Among authors: leroy bp. Eye (Lond). 2020 Jul;34(7):1158-1160. doi: 10.1038/s41433-020-0934-9. Epub 2020 May 19. Eye (Lond). 2020. PMID: 32427967 Free PMC article. No abstract available.
Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy.
Maguire AM, Bennett J, Aleman EM, Leroy BP, Aleman TS. Maguire AM, et al. Among authors: leroy bp. Mol Ther. 2021 Feb 3;29(2):442-463. doi: 10.1016/j.ymthe.2020.11.029. Epub 2020 Dec 3. Mol Ther. 2021. PMID: 33278565 Free PMC article. Review.
Genetic testing and diagnosis of inherited retinal diseases.
Lam BL, Leroy BP, Black G, Ong T, Yoon D, Trzupek K. Lam BL, et al. Among authors: leroy bp. Orphanet J Rare Dis. 2021 Dec 14;16(1):514. doi: 10.1186/s13023-021-02145-0. Orphanet J Rare Dis. 2021. PMID: 34906171 Free PMC article. Review.
The corneoscleral shape in Marfan syndrome.
Vanhonsebrouck E, Consejo A, Coucke PJ, Leroy BP, Kreps EO. Vanhonsebrouck E, et al. Among authors: leroy bp. Acta Ophthalmol. 2021 Jun;99(4):405-410. doi: 10.1111/aos.14636. Epub 2020 Sep 30. Acta Ophthalmol. 2021. PMID: 32996688 Free article.
Gene Therapy for Inherited Retinal Disease: Long-Term Durability of Effect.
Leroy BP, Fischer MD, Flannery JG, MacLaren RE, Dalkara D, Scholl HPN, Chung DC, Spera C, Viriato D, Banhazi J. Leroy BP, et al. Ophthalmic Res. 2023;66(1):179-196. doi: 10.1159/000526317. Epub 2022 Sep 14. Ophthalmic Res. 2023. PMID: 36103843 Free article. Review.
159 results