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Eine Ebene höherGruppiert nach: Dokumententyp |
Veröffentlichungsdatum
Anzahl der Publikationen: 11
2024
Yu-Wai-Man, Patrick; Carelli, Valerio; Newman, Nancy J.; Silva, Magda Joana; Linden, Aki; Van Stavern, Gregory; Szaflik, Jacek P.; Banik, Rudrani; Lubinski, Wojciech; Pemp, Berthold; Liao, Yaping Joyce; Subramanian, Prem S.; Misiuk-Hojlo, Marta; Newman, Steven; Castillo, Lorena; Kociecki, Jaroslaw; Levin, Marc H.; Munoz-Negrete, Francisco Jose; Yagan, Ali; Cherninkova, Sylvia; Katz, David; Meunier, Audrey; Votruba, Marcela; Korwin, Magdalena; Dziedziak, Jacek; Jurkute, Neringa; Harvey, Joshua P.; La Morgia, Chiara; Priglinger, Claudia; Lloria, Xavier; Tomasso, Livia und Klopstock, Thomas
(2024):
Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial.
In: Cell Reports Medicine, Bd. 5, Nr. 3, 101437
[PDF, 3MB]
2023
Vignal-Clermont, Catherine; Yu-Wai-Man, Patrick; Newman, Nancy J.; Carelli, Valerio; Moster, Mark L.; Biousse, Valerie; Subramanian, Prem S.; Wang, An-Guor; Donahue, Sean P.; Leroy, Bart P.; Sadun, Alfredo A.; Klopstock, Thomas; Sergott, Robert C.; Fernandez, Gema Rebolleda; Chwalisz, Bart K.; Banik, Rudrani; Taiel, Magali; Roux, Michel und Sahel, Jose-Alain
(2023):
Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy.
In: American Journal of Ophthalmology, Bd. 249: S. 108-125
[PDF, 1MB]
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]
Aytulun, Aykut; Cruz-Herranz, Andres; Aktas, Orhan; Balcer, Laura J.; Balk, Lisanne; Barboni, Piero; Blanco, Augusto Azuara; Calabresi, Peter A.; Costello, Fiona; Sanchez-Dalmau, Bernardo; DeBuc, Delia Cabrera; Feltgen, Nicolas; Finger, Robert P.; Frederiksen, Jette Lautrup; Frohman, Elliot; Frohman, Teresa; Garway-Heath, David; Gabilondo, Inigo; Graves, Jennifer S.; Green, Ari J.; Hartung, Hans-Peter; Havla, Joachim; Holz, Frank G.; Imitola, Jaime; Kenney, Rachel; Klistorner, Alexander; Knier, Benjamin; Korn, Thomas; Kolbe, Scott; Kraemer, Julia; Lagreze, Wolf A.; Leocani, Letizia; Maier, Oliver; Martinez-Lapiscina, Elena H.; Meuth, Sven; Outteryck, Olivier; Paul, Friedemann; Petzold, Axel; Pihl-Jensen, Gorm; Preiningerova, Jana Lizrova; Rebolleda, Gema; Ringelstein, Marius; Saidha, Shiv; Schippling, Sven; Schuman, Joel S.; Sergott, Robert C.; Toosy, Ahmed; Villoslada, Pablo; Wolf, Sebastian; Yeh, E. Ann; Yu-Wai-Man, Patrick; Zimmermann, Hanna G.; Brandt, Alexander U. und Albrecht, Philipp
(2021):
APOSTEL 2.0 Recommendations for Reporting Quantitative Optical Coherence Tomography Studies.
In: Neurology, Bd. 97, Nr. 2: S. 68-79
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]
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]
Biousse, Valerie; Newman, Nancy J.; Yu-Wai-Man, Patrick; Carelli, Valerio; Moster, Mark L.; Vignal-Clermont, Catherine; Klopstock, Thomas; Sadun, Alfredo A.; Sergott, Robert C.; Hage, Rabih; Esposti, Simona; La Morgia, Chiara; Priglinger, Claudia; Karanja, Rustum; Blouin, Laure; Taiel, Magali und Sahel, Jose-Alain
(2021):
Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study.
In: Journal of Neuro-Ophthalmology, Bd. 41, Nr. 3: S. 309-315
[PDF, 235kB]
2020
Yu-Wai-Man, Patrick; Newman, Nancy J.; Carelli, Valerio; Moster, Mark L.; Biousse, Valerie; Sadun, Alfredo A.; Klopstock, Thomas; Vignal-Clermont, Catherine; Sergott, Robert C.; Rudolph, Gunther; La Morgia, Chiara; Karanjia, Rustum; Taiel, Magali; Blouin, Laure; Burguiere, Pierre; Smits, Gerard; Chevalier, Caroline; Masonson, Harvey; Salermo, Yordak; Katz, Barrett; Picaud, Serge; Calkins, David J. und Sahel, Jose-Alain
(2020):
Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy.
In: Science Translational Medicine, Bd. 12, Nr. 573, eaaz7423
2016
Yu-Wai-Man, Patrick; Hudson, Gavin; Klopstock, Thomas und Chinnery, Patrick F.
(2016):
Reply: Parsing the differences in affected with LHON: genetic versus environmental triggers of disease conversion.
In: Brain, Bd. 139, e18
2014
Dimitriadis, Konstantin; Leonhardt, Miriam; Yu-Wai-Man, Patrick; Kirkman, Matthew Anthony; Korsten, Alex; De Coo, Irenaeus F.; Chinnery, Patrick Francis und Klopstock, Thomas
(2014):
Leber's hereditary optic neuropathy with late disease onset: clinical and molecular characteristics of 20 patients.
In: Orphanet Journal of Rare Diseases
9:158
[PDF, 200kB]