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Page 1
Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation.
Carelli V, Newman NJ, Yu-Wai-Man P, Biousse V, Moster ML, Subramanian PS, Vignal-Clermont C, Wang AG, Donahue SP, Leroy BP, Sergott RC, Klopstock T, Sadun AA, Rebolleda Fernández G, Chwalisz BK, Banik R, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA; the LHON Study Group. Carelli V, et al. Among authors: debusk aa. Ophthalmol Ther. 2023 Feb;12(1):401-429. doi: 10.1007/s40123-022-00611-x. Epub 2022 Nov 30. Ophthalmol Ther. 2023. PMID: 36449262 Free PMC article.
Gene therapy in optic nerve disease.
DeBusk A, Moster ML. DeBusk A, et al. Curr Opin Ophthalmol. 2018 May;29(3):234-238. doi: 10.1097/ICU.0000000000000473. Curr Opin Ophthalmol. 2018. PMID: 29538182 Review.
Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset.
Newman NJ, Yu-Wai-Man P, Carelli V, Moster ML, Biousse V, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Barboni P, DeBusk AA, Girmens JF, Rudolph G, Karanjia R, Taiel M, Blouin L, Smits G, Katz B, Sahel JA; LHON Study Group. Newman NJ, et al. Among authors: debusk aa. Ophthalmology. 2021 May;128(5):649-660. doi: 10.1016/j.ophtha.2020.12.012. Epub 2021 Jan 12. Ophthalmology. 2021. PMID: 33451738 Free article. Clinical Trial.
Natural history of patients with Leber hereditary optic neuropathy-results from the REALITY study.
Yu-Wai-Man P, Newman NJ, Carelli V, La Morgia C, Biousse V, Bandello FM, Clermont CV, Campillo LC, Leruez S, Moster ML, Cestari DM, Foroozan R, Sadun A, Karanjia R, Jurkute N, Blouin L, Taiel M, Sahel JA; LHON REALITY Study Group. Yu-Wai-Man P, et al. Eye (Lond). 2022 Apr;36(4):818-826. doi: 10.1038/s41433-021-01535-9. Epub 2021 Apr 28. Eye (Lond). 2022. PMID: 33911213 Free PMC article.
Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison.
Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA. Newman NJ, et al. Among authors: debusk aa. Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021. Front Neurol. 2021. PMID: 34108929 Free PMC article.
Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies.
Moster ML, Sergott RC, Newman NJ, Yu-Wai-Man P, Carelli V, Bryan MS, Smits G, Biousse V, Vignal-Clermont C, Klopstock T, Sadun AA, DeBusk AA, Carbonelli M, Hage R, Priglinger S, Karanjia R, Blouin L, Taiel M, Katz B, Sahel JA; LHON study group. Moster ML, et al. Among authors: debusk aa. J Neuroophthalmol. 2021 Sep 1;41(3):298-308. doi: 10.1097/WNO.0000000000001316. J Neuroophthalmol. 2021. PMID: 34310464 Free PMC article. Clinical Trial.
Mismatch in Supply and Demand for Neuro-Ophthalmic Care.
DeBusk A, Subramanian PS, Scannell Bryan M, Moster ML, Calvert PC, Frohman LP. DeBusk A, et al. J Neuroophthalmol. 2022 Mar 1;42(1):62-67. doi: 10.1097/WNO.0000000000001214. Epub 2021 Mar 23. J Neuroophthalmol. 2022. PMID: 33770009
Occam versus Hickam.
Cobbs L, Murchison AP, DeBusk A, Bilyk JR. Cobbs L, et al. Surv Ophthalmol. 2022 Nov-Dec;67(6):1717-1722. doi: 10.1016/j.survophthal.2022.06.001. Epub 2022 Jun 17. Surv Ophthalmol. 2022. PMID: 35718026
Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy.
Newman NJ, Yu-Wai-Man P, Subramanian PS, Moster ML, Wang AG, Donahue SP, Leroy BP, Carelli V, Biousse V, Vignal-Clermont C, Sergott RC, Sadun AA, Rebolleda Fernández G, Chwalisz BK, Banik R, Bazin F, Roux M, Cox ED, Taiel M, Sahel JA; LHON REFLECT Study Group. Newman NJ, et al. Brain. 2023 Apr 19;146(4):1328-1341. doi: 10.1093/brain/awac421. Brain. 2023. PMID: 36350566 Free PMC article. Clinical Trial.