| Identifier |
20230314_nanos_posters_274 |
| Title |
Indirect Comparison of Lenadogene Nolparvovec Gene Therapy versus Natural History in m.11778G>A MT-ND4 Leber Hereditary Optic Neuropathy Patients |
| Creator |
Mark Moster; Nancy Newman; Valerio Carelli; Patrick Yu-Wai-Man; Valerie Biousse; Prem Subramanian; Catherine Vignal-Clermont; An-Guor Wang; Sean Donahue; Bart Leroy; Robert Sergott; Thomas Klopstock; Alfredo Sadun; Gema Rebolleda Fernandez; Bart Chwalisz; Rudrani Banik; Magali Taiel; José-Alain Sahel |
| Affiliation |
(MM) (RS) Wills Eye Hospital and Thomas Jefferson University; (NN) Departments of Ophthalmology, Neurology, and Neurological Surgery, Emory University School of Medicine, Atlanta, GA; (VC) IRCCS Istituto delle Scienze Neurologiche di Bologna; (PY) Department of Clinical Neurosciences, University of Cambridge; (VB) Departments of Ophthalmology and Neurology, Emory University School of Medicine, Atlanta, GA; (PS) University of Colorado Anschutz Medical Campus; (CV) Rothschild Foundation Hospital; (AW) Taipei Veterans General Hospital; (SD) Vanderbilt University Medical Center; (BL) Ghent University Hospital; (TK) Friedrich-Baur-Institut; (AS) Doheny Eye Institute, UCLA School of Medicine; (GRF) Alcala University; (BC) Massachusetts General Hospital; (RB) Icahn School of Medicine at Mount Sinai' (MT) GenSight Biologics; (JS) Institut de la Vision |
| Subject |
Optic Neuropathy; Genetic Disease |
| Description |
Lenadogene nolparvovec is a promising gene therapy for patients with Leber hereditary optic neuropathy (LHON) due to the m.11778G>A MT-ND4 mutation. The purpose of this analysis was to compare the visual acuity of MT-ND4 LHON patients treated with lenadogene nolparvovec in clinical studies to the spontaneous evolution of visual acuity in an external control group of untreated MT-ND4 LHON patients. A previous analysis was performed on treated patients from three phase 3 studies. This updated analysis incorporates a fourth study, REFLECT, in which patients received lenadogene nolparvovec unilaterally or bilaterally. |
| Date |
2023-03-14 |
| Language |
eng |
| Format |
application/pdf |
| Type |
Text |
| Source |
2023 North American Neuro-Ophthalmology Society Annual Meeting |
| Relation is Part of |
NANOS Annual Meeting 2023: Poster Session II: Neuro-Ophthalmic Disorders of Neurologic and Systemic Diseases |
| Collection |
Neuro-ophthalmology Virtual Education Library: NOVEL http://NOVEL.utah.edu |
| Publisher |
Spencer S. Eccles Health Sciences Library, University of Utah |
| Holding Institution |
North American Neuro-Ophthalmology Association. NANOS Executive Office 5841 Cedar Lake Road, Suite 204, Minneapolis, MN 55416 |
| Rights Management |
Copyright 2023. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright |
| ARK |
ark:/87278/s6v9kn41 |
| Setname |
ehsl_novel_nam |
| ID |
2335479 |
| Reference URL |
https://collections.lib.utah.edu/ark:/87278/s6v9kn41 |
