| Identifier |
20230314_nanos_posters_278 |
| Title |
Treatment with Lenadogene Nolparvovec Gene Therapy Results in Sustained Visual Improvement in m.11778G>A MT-ND4 LHON Patients: the RESTORE Study |
| Creator |
Patrick Yu-Wai-Man; Nancy Newman; Valerie Biousse; Valerio Carelli; Mark Moster; Catherine Vignal-Clermont; Thomas Klopstock; Alfredo Sadun; Robert Sergott; Magali Taiel; Josﾎ-Alain Sahel |
| Affiliation |
(PYWM) Department of Clinical Neurosciences, University of Cambridge; (NN) Departments of Ophthalmology, Neurology, and Neurological Surgery, Emory University School of Medicine, Atlanta, GA; (VB) Departments of Ophthalmology and Neurology, Emory University School of Medicine, Atlanta, GA; (VC) IRCCS Instituto delle Scienze Neurologiche di Bologna, Italy; (MM) Wills Eye Hospital and Thomas Jefferson University; (CVC) Rothschild Foundation Hospital; (TK) Friedrich-Baur-Institut; (AS) Doheny Eye Institute, UCLA School of Medicine; (MT) GenSight Biologics; (10) Institut de la Vision |
| Subject |
Optic Neuropathy; Genetic Disease |
| Description |
Two phase 3 clinical studies, RESCUE and REVERSE, assessed the efficacy and safety of an intravitreal injection of lenadogene nolparvovec gene therapy in patients with Leber hereditary optic neuropathy (LHON) due to the m.11778G>A mitochondrial DNA mutation in MT-ND4. Recruited patients were followed up for a period of 5 years posttreatment as part of the long-term RESTORE study. |
| 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/s6dcytm8 |
| Setname |
ehsl_novel_nam |
| ID |
2335483 |
| Reference URL |
https://collections.lib.utah.edu/ark:/87278/s6dcytm8 |
