Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial.

MacLaren, Robert E; Fischer, M Dominik; Gow, James A; Lam, Byron L; Sankila, Eeva-Marja K; Girach, Aniz; Panda, Sushil; Yoon, Dan; Zhao, Guolin; Pennesi, Mark E

MacLaren, Robert E; Fischer, M Dominik; Gow, James A; Lam, Byron L; Sankila, Eeva-Marja K; Girach, Aniz; Panda, Sushil; Yoon, Dan; Zhao, Guolin; Pennesi, Mark E.

Affiliation

MacLaren RE; Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, University of Oxford, Oxford, UK. enquiries@eye.ox.ac.uk.
Fischer MD; Oxford University Hospitals NIHR Biomedical Research Centre, Oxford, UK. enquiries@eye.ox.ac.uk.
Gow JA; Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK. enquiries@eye.ox.ac.uk.
Lam BL; University Eye Hospital Tübingen, Center for Ophthalmology, Tübingen, Germany.
Sankila EK; Biogen, Cambridge, MA, USA.
Girach A; Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA.
Panda S; Helsinki University Eye Hospital, Helsinki, Finland.
Yoon D; Formerly of Nightstar Therapeutics, London, UK.
Zhao G; Biogen, Cambridge, MA, USA.
Pennesi ME; Biogen, Cambridge, MA, USA.

Nat Med ; 29(10): 2464-2472, 2023 10.

Article in En | MEDLINE | ID: mdl-37814062

Subject(s)

Choroideremia; Diabetic Retinopathy; Male; Humans; Adult; Choroideremia/genetics; Choroideremia/therapy; Visual Acuity; Genetic Therapy/adverse effects; Genetic Therapy/methods; Retina

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Choroideremia / Diabetic Retinopathy Type of study: Clinical_trials Limits: Adult / Humans / Male Language: En Journal: Nat Med Journal subject: BIOLOGIA MOLECULAR / MEDICINA Year: 2023 Document type: Article Affiliation country: United kingdom Country of publication: United States

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