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: Evaluation of rAAV2/2-ND4 Gene Therapy Efficacy in Leber Hereditary Optic Neuropathy Using an External Control Group of Untreated Patients

Researchers

Presenter

  • Nancy Newman

Principal Investigators

  • Patrick Yu-Wai-Man

  • Valerio Carelli

  • Mark Moster

  • Catherine Vignal-Clermont

  • Alfredo Sadun

  • Chiara La Morgia

  • Francesco Bandello

  • Lorena Castillo Campillo

  • Stphanie Leruez

  • Dean Cestari

  • Rod Foroozan

  • Rustum Karanjia

  • Laure Blouin

  • Michel Roux

  • Magali Taiel

  • Jose-Alain Sahel

Medical Centers

  • Ajou University school of Medicine

  • Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK. Cambridge Eye Unit, Addenbrooke’s Hospital, Cambridge University Hospitals, Cambridge, UK. Moorfields Eye Hospital, London, UK. UCL Institute of Ophthalmology, University College London, London, UK

  • University of Bologna

  • Neuro-Ophthalmology -Wills Eye Hospital

  • Foundation Ophtalmologique A De Rothschild

  • UCLA,

  • Department of Ophthalmology Vita-Salute San Raffaele University

  • IRCCS San Raffaele Scientific Institute

  • Institut Català de Retina

  • CHU Angers - Hôpital Hôtel Dieu

  • Massachusetts Eye and Ear Infirmary

  • Alkek Eye Center

  • Doheny Eye Center UCLA

  • GenSight Biologics

  • Department of Pathology, University of Pittsburgh School of Medicine

  • Foundation Ophtalmologique A De Rothschild

  • Sorbonne Université, INSERM, CNRS, Institut de la Vision

  • CHNO des Quinze-Vingts, Institut Hospitalo-Universitaire FOReSIGHT

Locations

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Ophthalmology (OP)

  • Genetic Disorder

Disease

  • Leber optic atrophy

Biomarkers

  • N/A

Drug/Treatment

  • Lenadogene nolparvovec

Outcome

  • N/A

Study Design

  • N/A

Phase

  • III

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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