Study design and baseline characteristics for the reflect gene therapy trial ofm.11778g>A/ND4-LHON

Autor: Nancy J Newman, Valérie Biousse, José-Alain Sahel, Patrick Yu-Wai-Man, Sean Donahue, Gema Rebolleda, Prem S Subramanian, Bart P Leroy, Alfredo A Sadun, Robert C Sergott, Catherine Vignal-Clermont, Bart K Chwalisz, Mark Moster, An-Guor Wang, Valerio Carelli, Rudrani Banik, Fabienne Bazin, Eric Cox, Michel Roux, Magali Taiel, Amore Giulia, Anand Shweta, Banik Rudrani, Barboni Piero, Biousse Valérie, Boston Hayley, Burale Asma, Carbonelli Michele, Carelli Valerio, Chen Celia, Cheng Hui-Chen, Cho Steve, K Chwalisz Bart, Contin Manuela, D’Agati Pietro, A DeBusk Adam, De Zaeytijd Julie, Dobbs Jannah, P Donahue Sean, DuBois Lindreth, Esposti Simona, Fernandes Filho Alcides, Fortin Elizabeth, Gangaputra Sapna, Gibbs Deborah, Girmens Jean François, Hage Rabih, A Haller Julia, Heilweil Gad, Hubbard III GeorgeBaker, Hwang Jeong-Min, Jaumendreu Urquijo Laia, Jurkute Neringa, Karanjia Rustum, Khemliche Wahiba, La Morgia Chiara, P Leroy Bart, Massini Maria, Mathias Marc, A Memon Muhammad, Mohamed Susan, L Moster Mark, J Muñoz NegreteFrancisco, J Newman Nancy, O’Keefe Ghazala, Patel Shriji, Pecen Paula, H Peragallo Jason, Plaine Lise, Preston Mary, Rebolleda Fernández Gema, Romagnoli Martina, A Sadun Alfredo, A Sahel José, SantaMaria Melissa, C Sergott Robert, S Subramanian Prem, Sun Chuanbin, Tai Katy, Tollis Heather, Tsui Irena, R Tucker William, Vignal-Clermont Catherine, Wang An-Guor, Wilkins Saige, Yu-Wai-Man Patrick

Publikováno v: BMJ Open Ophthalmology, Vol 7, Iss 1 (2022)

Objective REFLECT is the first randomised, double-masked, placebo-controlled multicentre phase 3 clinical trial that evaluated the efficacy and safety of bilateral intravitreal (IVT) injection of lenadogene nolparvovec in subjects with Leber heredita

Externí odkaz: https://doaj.org/article/c3ae820deb6448238032cfa7c2f4a8d2

Leber Hereditary Optic Neuropathy: Review of Treatment and Management

Autor: Rabih Hage, Catherine Vignal-Clermont

Publikováno v: Frontiers in Neurology, Vol 12 (2021)

Leber hereditary optic neuropathy (LHON) is a maternally inherited mitochondrial disease that specifically targets the retinal ganglion cells by reducing their ability to produce enough energy to sustain. The mutations of the mitochondrial DNA that c

Externí odkaz: https://doaj.org/article/2574dce574cf48dcbf6989ef95991f11

Generalization of ocular myasthenia gravis 10 years after onset

Autor: Antoine Guéguen, Benoit Hijazi, Kevin Zuber, Romain Deschamps, Cedric Lamirel, Catherine Vignal-Clermont, Olivier Gout

Publikováno v: Journal of Neurology. 269:6597-6604

Generalization of ocular myasthenia gravis (OMG) represents a pejorative evolution, and no validated generalization-prevention strategy exists. The study aimed to determine the percentage of patients with OMG generalization and identify factors predi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::757433a3d7d2ea2767fc810587b47c19
https://doi.org/10.1007/s00415-022-11316-3

Incidence of Eye Trauma in Children Associated With Foam Bullets or Foam Darts From Nonpowder Guns

Autor: Alexandre Dentel, Etienne Boulanger, Catherine Vignal-Clermont, Georges Caputo, Gilles C. Martin

Publikováno v: JAMA Ophthalmology.

This case series estimates the annual incidence of pediatric eye injuries associated with recreational use of nonpowder guns at an ophthalmologic emergency department in France.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::7df0222a0ffb5350c88131e8129cd46e
https://doi.org/10.1001/jamaophthalmol.2023.1464

Long-term Follow-up of m.11778G>A MT-ND4-LHON Patients Treated with Lenadogene Nolparvovec Ocular Gene Therapy: the RESTORE Study (S12.004)

Autor: Valerie Biousse, Nancy Newman, Patrick Yu-Wai-Man, Valerio Carelli, Mark Moster, Catherine Vignal-Clermont, Thomas Klopstock, Alfredo Sadun, Robert Sergott, Magali Taiel, Jose-Alain Sahel

Publikováno v: Monday, April 24.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::bc1ecb0e5b56d1d32338f5319d01a924
https://doi.org/10.1212/wnl.0000000000202279