Carelli, Valerio http://orcid.org/0000-0003-4923-6404
Newman, Nancy J.
Yu-Wai-Man, Patrick
Biousse, Valerie
Moster, Mark L.
Subramanian, Prem S.
Vignal-Clermont, Catherine
Wang, An-Guor
Donahue, Sean P.
Leroy, Bart P.
Sergott, Robert C.
Klopstock, Thomas
Sadun, Alfredo A.
Rebolleda Fernández, Gema
Chwalisz, Bart K.
Banik, Rudrani
Girmens, Jean François
La Morgia, Chiara
DeBusk, Adam A.
Jurkute, Neringa
Priglinger, Claudia
Karanjia, Rustum
Josse, Constant
Salzmann, Julie
Montestruc, François
Roux, Michel
Taiel, Magali
Sahel, José-Alain
,
Carelli, Valerio
Barboni, Piero
Carbonelli, Michele
Di Vito, Lidia
Amore, Giulia
Contin, Manuela
Mohamed, Susan
La Morgia, Chiara
Silvestri, Sara
D’Agati, Pietro
Newman, Nancy J
Biousse, Valérie
Hubbard, George Baker
O’Keefe, Ghazala
Hendrick, Andrew M.
Dattilo, Michael
Peragallo, Jason H.
Hawy, Eman
DuBois, Lindreth
Gibbs, Deborah
Filho, Alcides Fernandes
Dobbs, Jannah
Yu-Wai-Man, Patrick
Acheson, James
Boston, Hayley
Eleftheriadou, Maria
Esposti, Simona
Gemenetzi, Maria
Leitch-Devlin, Lauren
Tucker, William R.
Jurkute, Neringa
Burale, Asma
Anand, Shweta
Memon, Muhammad A.
Hussain, Rima
Jorany, Rasha
Sheel, Priyansha
Moster, Mark L.
Sergott, Robert C.
SantaMaria, Melissa
Tollis, Heather
DeBusk, Adam A
Haller, Julia A.
Massini, Maria
Subramanian, Prem S.
Pecen, Paula
Mathias, Marc
Preston, Mary
Cho, Steve
Sahel, José A.
Vignal-Clermont, Catherine
Girmens, Jean François
Hage, Rabih
Plaine, Lise
Khemliche, Wahiba
Wang, An-Guor
Cheng, Hui-Chen
Chen, Celia
Hwang, Jeong-Min
Sun, Chuanbin
Donahue, Sean
Patel, Shriji
Gangaputra, Sapna
Barrett, Megan
Ruark, Scott
Wilkins, Saige
Leroy, Bart P.
De Zaeytijd, Julie
Van Cauwenbergh, Caroline
Verhauwen, Hilde
Klopstock, Thomas
Catarino, Claudia B.
Priglinger, Claudia
Priglinger, Siegfried
Rudolph, Günther
von Livonius Bettina, Stephan Thurau
Muth, Daniel
Wolf, Armin
Al-Tamami, Jasmina
Pressler, Angelika
Schertler, Cosima
Hildebrandt, Martin
Neuenhahn, Michael
Sadun, Alfredo A.
Heilweil, Gad
Karanjia, Rustum
Tsui, Irena
Rebolleda Fernández, Gema
Urquijo, Laia Jaumendreu
Negrete Muñoz, Francisco J.
Fortin, Elizabeth
Chwalisz, Bart K.
Cestari, Dean
Banik, Rudrani
Tai, Katy
Castillo, Lorena
Garcia, Virginia
Bandello, Antonio Morilla Francesco
Barboni, Piero
Cascavilla, Maria Lucia
Battista, Marco
Calcagno, Francesca
Pina, Adelaide
Leruez, Stéphanie
Forooza, Rod
Clinical trials referenced in this document:
Documents that mention this clinical trial
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
https://doi.org/10.1007/s40123-022-00611-x
Documents that mention this clinical trial
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
https://doi.org/10.1007/s40123-022-00611-x
Documents that mention this clinical trial
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
https://doi.org/10.1007/s40123-022-00611-x
Documents that mention this clinical trial
Study design and baseline characteristics for the reflect gene therapy trial ofm.11778g>A/<i>ND4</i>-LHON
https://doi.org/10.1136/bmjophth-2022-001158
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
https://doi.org/10.1007/s40123-022-00611-x
Documents that mention this clinical trial
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
https://doi.org/10.1007/s40123-022-00611-x
Funding for this research was provided by:
GenSight Biologics
Italian Ministry of Health (RF-2018-12366703)
Italian Ministry of University and Research (20172T2MHH)
Telethon-Italy (GGP20115)
patients’ organizations MITOCON and IFOND, and patients’ donations
NIH/NEI (P30-EY06360)
NIH/NEI (P30-EY06360)
NIH/NINDS (RO1NSO89694)
NIH/NINDS (RO1NSO89694)
UK National Institute of Health Research (NIHR301696)
UK Medical Research Council (G1002570)
Fight for Sight UK
Isaac Newton Trust
Moorfields Eye Charity (GR001376)
Addenbrooke’s Charitable Trust
National Eye Research Centre
International Foundation for Optic Nerve Disease
NIHR as part of the Rare Diseases Translational Research Collaboration
NIHR Cambridge Biomedical Research Centre (BRC-1215-20014)
National Institute for Health Research Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology
Research Foundation - Flanders, Belgium (Senior Clinical Investigator 1803821N)
Concerted Research Action of the Special Research Fund Ghent University (BOF20/GOA/023)
German Network for Mitochondrial Disorders (mitoNET, 01GM1906A)
E-Rare project GENOMIT (01GM1920B)
Agence Nationale de la Recherche, Programme Investissements d'Avenir (ANR-18-IAHU-0001)
LabEx LIFESENSES (ANR-10-LABX- 65)
Article History
Received: 3 October 2022
Accepted: 28 October 2022
First Online: 30 November 2022