Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy

An Economic Evaluation of Voretigene Neparvovec for the Treatment of Biallelic RPE65-Mediated Inherited Retinal Dystrophies in the UK

(2020) Daniel Viriato et al.   ADVANCES IN THERAPY

Luxturna: FDA documents reveal the value of a costly gene therapy

(2019) Jonathan J. Darrow   DRUG DISCOVERY TODAY

Cost-effectiveness of Voretigene Neparvovec-rzyl vs Standard Care for RPE65-Mediated Inherited Retinal Disease

(2019) Scott Johnson et al.   JAMA Ophthalmology

How long does gene therapy last? 4-year follow-up of phase 3 voretigene neparvovec trial in RPE65-associated LCA/inherited retinal disease

(2019) Arlene V. Drack et al.   JOURNAL OF AAPOS

Assessment of the Electronic Retinal Implant Alpha AMS in Restoring Vision to Blind Patients with End-Stage Retinitis Pigmentosa

(2018) Thomas L. Edwards et al.   OPHTHALMOLOGY

PSY28 - EPIDEMIOLOGY OF RPE65 GENE MUTATION RELATED INHERITED RETINAL DYSTROPHIES: A SYSTEMATIC LITERATURE REVIEW

(2018) C. Aouadj et al.   VALUE IN HEALTH

Cost Utility of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease

(2018) Marita Zimmermann et al.   VALUE IN HEALTH

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

(2016) Jean Bennett et al.   LANCET

Comprehensive genotyping reveals RPE65 as the most frequently mutated gene in Leber congenital amaurosis in Denmark

(2015) Galuh D N Astuti et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Advancing Therapeutic Strategies for Inherited Retinal Degeneration: Recommendations From the Monaciano Symposium

(2015) D. A. Thompson et al.   INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET