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.

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Title: 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.
Authors: Carelli, Valerio1,2 (AUTHOR) valerio.carelli@unibo.it, Newman, Nancy J.3,4,5 (AUTHOR), Yu-Wai-Man, Patrick6,7,8,9 (AUTHOR), Biousse, Valerie3,4,5 (AUTHOR), Moster, Mark L.10,11 (AUTHOR), Subramanian, Prem S.12 (AUTHOR), Vignal-Clermont, Catherine13,14 (AUTHOR), Wang, An-Guor15 (AUTHOR), Donahue, Sean P.16 (AUTHOR), Leroy, Bart P.17,18 (AUTHOR), Sergott, Robert C.10,11 (AUTHOR), Klopstock, Thomas19,20,21 (AUTHOR), Sadun, Alfredo A.22 (AUTHOR), Rebolleda Fernández, Gema23 (AUTHOR), Chwalisz, Bart K.24 (AUTHOR), Banik, Rudrani25 (AUTHOR), Girmens, Jean François14 (AUTHOR), La Morgia, Chiara1,2 (AUTHOR), DeBusk, Adam A.10,11 (AUTHOR), Jurkute, Neringa8,9,26 (AUTHOR)
Source: Ophthalmology & Therapy. Feb2023, Vol. 12 Issue 1, p401-429. 29p.
Database: Academic Search Ultimate
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ISSN:21938245
DOI:10.1007/s40123-022-00611-x