Anzahl der Publikationen: 5
2024
Yu-Wai-Man, Patrick; Newman, Nancy J.; Biousse, Valérie; Carelli, Valerio; Moster, Mark L.; Vignal-Clermont, Catherine; Klopstock, Thomas ORCID: https://orcid.org/0000-0003-2805-4652; Sadun, Alfredo A.; Sergott, Robert C.; Hage, Rabih; Degli Esposti, Simona; La Morgia, Chiara; Priglinger, Claudia; Karanja, Rustum; Taiel, Magali; Sahel, José-Alain; Barboni, Piero; Carbonelli, Michele; Di Vito, Lidia; Amore, Giulia; Contin, Manuela; Mohamed, Susan; Silvestri, Sara; Baker Hubbard, George; Hendrick, Andrew M.; Dattilo, Michael; Peragallo, Jason H.; Hawy, Eman; DuBois, Lindreth; Gibbs, Deborah; Fernandes Filho, Alcides; Dobbs, Jannah; Aung, Andre; Acheson, James; Boston, Hayley; Eleftheriadou, Maria; Gemenetzi, Maria; Leitch-Devlin, Lauren; Tucker, William R.; Jurkute, Neringa; Burale, Asma; DeBusk, Adam A.; Haller, Julia A.; Massini, Maria; SantaMaria, Melissa; Tollis, Heather; Girmens, Jean-François; Plaine, Lise; Khemliche, Wahiba; Catarino, Claudia B.; Priglinger, Siegfried ORCID: https://orcid.org/0000-0002-5580-612X; Rudolph, Günther; Thurau, Stephan R.; Livonius, Bettina von; Muth, Daniel; Wolf, Armin; Al-Tamami, Jasmina; Pressler, Angelika; Schertler, Cosima; Hildebrandt, Martin; Neuenhahn, Michael; Heilweil, Gad und Tsui, Irena
(2024):
Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.
In: JAMA Ophthalmology [Forthcoming]
2022
Carelli, Valerio; 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.; Fernandez, Gema Rebolleda; Chwalisz, Bart K.; Banik, Rudrani; Girmens, Jean Francois; La Morgia, Chiara; DeBusk, Adam A.; Jurkute, Neringa; Priglinger, Claudia; Karanjia, Rustum; Josse, Constant; Salzmann, Julie; Montestruc, Francois; Roux, Michel; Taiel, Magali; Sahel, Jose-Alain und Group, The Lhon Study
(2022):
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.
In: Ophthalmology and Therapy, Bd. 12, Nr. 1: S. 401-429
[PDF, 719kB]
2021
Newman, Nancy J.; Yu-Wai-Man, Patrick; Carelli, Valerio; Biousse, Valerie; Moster, Mark L.; Vignal-Clermont, Catherine; Sergott, Robert C.; Klopstock, Thomas; Sadun, Alfredo A.; Girmens, Jean-Francois; La Morgia, Chiara; DeBusk, Adam A.; Jurkute, Neringa; Priglinger, Claudia; Karanjia, Rustum; Josse, Constant; Salzmann, Julie; Montestruc, Francois; Roux, Michel; Taiel, Magali und Sahel, Jose-Alain
(24. Mai 2021):
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.
In: Frontiers in Neurology, Bd. 12, 662838
[PDF, 592kB]
Moster, Mark L.; Sergott, Robert C.; Newman, Nancy J.; Yu-Wai-Man, Patrick; Carelli, Valerio; Bryan, Molly Scannell; Smits, Gerard; Biousse, Valerie; Vignal-Clermont, Catherine; Klopstock, Thomas ORCID: https://orcid.org/0000-0003-2805-4652; Sadun, Alfredo A.; DeBusk, Adam A.; Carbonelli, Michele; Hage, Rabih; Priglinger, Siegfried ORCID: https://orcid.org/0000-0002-5580-612X; Karanjia, Rustum; Blouin, Laure; Taiel, Magali; Katz, Barrett und Sahel, Jose Alain
(2021):
Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies.
In: Journal of Neuro-Ophthalmology, Bd. 41, Nr. 3: S. 298-308
[PDF, 363kB]
Newman, Nancy J.; Yu-Wai-Man, Patrick; Carelli, Valerio; Moster, Mark L.; Biousse, Valerie; Vignal-Clermont, Catherine; Sergott, Robert C.; Klopstock, Thomas; Sadun, Alfredo A.; Barboni, Piero; DeBusk, Adam A.; Girmens, Jean Francois; Rudolph, Guenther; Karanjia, Rustum; Taiel, Magali; Blouin, Laure; Smits, Gerard; Katz, Barrett und Sahel, Jose-Alain
(2021):
Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset.
In: Ophthalmology, Bd. 128, Nr. 5: S. 649-660
[PDF, 894kB]
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