Cargando…

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

IMPORTANCE: Voretigene neparvovec-rzyl, the first gene therapy approved by the US Food and Drug Administration, was approved for the treatment for RPE65-mediated inherited retinal disease (IRD) in December 2017. This gene therapy is associated with high up-front costs and high efficacy, although of...

Descripción completa

Detalles Bibliográficos
Autores principales:	Johnson, Scott, Buessing, Marric, O’Connell, Thomas, Pitluck, Sarah, Ciulla, Thomas A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Medical Association 2019
Materias:	Original Investigation
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6646972/ https://www.ncbi.nlm.nih.gov/pubmed/31318398 http://dx.doi.org/10.1001/jamaophthalmol.2019.2512

Ejemplares similares

Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation
por: Ameri, Hossein
Publicado: (2018)

Bilateral exudative retinal detachments after subretinal gene therapy with voretigene neparvovec-rzyl for RPE65 Leber Congenital Amaurosis
por: Lidder, Alcina K., et al.
Publicado: (2023)

Cost Effectiveness of Voretigene Neparvovec for RPE65-Mediated Inherited Retinal Degeneration in Germany
por: Uhrmann, Matthias Fritz, et al.
Publicado: (2020)

Estimation of impact of RPE65-mediated inherited retinal disease on quality of life and the potential benefits of gene therapy
por: Lloyd, Andrew, et al.
Publicado: (2019)

Retinal Pigment Epithelium Atrophy After Subretinal Voretigene Neparvovec-rzyl for RPE65-Related Disease: A 6-Month Follow-Up
por: Giansanti, Fabrizio, et al.
Publicado: (2022)

Voretigene neparvovec for inherited retinal dystrophy
Publicado: (2021)

An Economic Evaluation of Voretigene Neparvovec for the Treatment of Biallelic RPE65-Mediated Inherited Retinal Dystrophies in the UK
por: Viriato, Daniel, et al.
Publicado: (2020)

Cost-effectiveness of voretigene neparvovec in the treatment of patients with inherited retinal disease with RPE65 mutation in Switzerland
por: Bhadhuri, Arjun, et al.
Publicado: (2022)

Improved Rod Sensitivity as Assessed by Two-Color Dark-Adapted Perimetry in Patients With RPE65-Related Retinopathy Treated With Voretigene Neparvovec-rzyl
por: Ku, Cristy A., et al.
Publicado: (2023)

Visual function and retinal changes after voretigene neparvovec treatment in children with biallelic RPE65-related inherited retinal dystrophy
por: Testa, Francesco, et al.
Publicado: (2022)

Voretigene Neparvovec for the Treatment of RPE65-associated Retinal Dystrophy: Consensus and Recommendations from the Korea RPE65-IRD Consensus Paper Committee
por: Han, Jinu, et al.
Publicado: (2023)

Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center
por: Kiraly, Peter, et al.
Publicado: (2023)

Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence
por: Gao, Jie, et al.
Publicado: (2020)

Chorioretinal atrophy following voretigene neparvovec despite the presence of fundus autofluorescence
por: Kolesnikova, Masha, et al.
Publicado: (2022)

Voretigene Neparvovec and Gene Therapy for Leber’s Congenital Amaurosis: Review of Evidence to Date
por: Padhy, Srikanta Kumar, et al.
Publicado: (2020)

Spatial and temporal resolution of the photoreceptors rescue dynamics after treatment with voretigene neparvovec
por: Stingl, Krunoslav, et al.
Publicado: (2022)

Voretigene Neparvovec Gene Therapy in Clinical Practice: Treatment of the First Two Italian Pediatric Patients
por: Testa, Francesco, et al.
Publicado: (2021)

Assessing the Accuracy, Quality, and Readability of Patient Accessible Online Resources Regarding Ocular Gene Therapy and Voretigene Neparvovec
por: Davuluri, Swetha, et al.
Publicado: (2021)

Gene Therapy with Voretigene Neparvovec Improves Vision and Partially Restores Electrophysiological Function in Pre-School Children with Leber Congenital Amaurosis
por: Gerhardt, Maximilian J., et al.
Publicado: (2022)

RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy
por: Sodi, Andrea, et al.
Publicado: (2021)

Inherited Retinal Diseases Due to RPE65 Variants: From Genetic Diagnostic Management to Therapy
por: Aoun, Manar, et al.
Publicado: (2021)

Retinal Structure in RPE65-Associated Retinal Dystrophy
por: Kumaran, Neruban, et al.
Publicado: (2020)

A Molecular Genetic Analysis of RPE65-Associated Forms of Inherited Retinal Degenerations in the Russian Federation
por: Stepanova, Anna, et al.
Publicado: (2023)

Epidemiology of Mutations in the 65-kDa Retinal Pigment Epithelium (RPE65) Gene-Mediated Inherited Retinal Dystrophies: A Systematic Literature Review
por: Sallum, Juliana M. F., et al.
Publicado: (2022)

Clinical Features and Natural History in a Cohort of Chinese Patients with RPE65-Associated Inherited Retinal Dystrophy
por: Shi, Jie, et al.
Publicado: (2021)

Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision
por: Huygens, Simone A., et al.
Publicado: (2021)

Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)
por: Scholl, Hendrik P. N., et al.
Publicado: (2015)

A Comprehensive Study of the Retinal Phenotype of Rpe65-Deficient Dogs
por: Annear, Matthew J, et al.
Publicado: (2021)

Frequency of RPE65 Gene Mutation in Patients with Hereditary Retinal Dystrophy
por: Sinim Kahraman, Neslihan, et al.
Publicado: (2022)

Carrier frequency and incidence estimation of RPE65-associated inherited retinal diseases in East Asian population by population database-based analysis
por: Cho, Eun Hye, et al.
Publicado: (2022)

Development of a novel prediction model based on protein structure for identifying RPE65-associated inherited retinal disease (IRDs) of missense variants
por: Wu, Jiawen, et al.
Publicado: (2023)

Differential neuroglycan C expression during retinal degeneration in Rpe65(−/−) mice
por: Escher, Pascal, et al.
Publicado: (2008)

Novel mutations in RPE65 identified in consanguineous Pakistani families with retinal dystrophy
por: Kabir, Firoz, et al.
Publicado: (2013)

The clinical features of retinal disease due to a dominant mutation in RPE65
por: Hull, Sarah, et al.
Publicado: (2016)

Care Pathway of RPE65-Related Inherited Retinal Disorders from Early Symptoms to Genetic Counseling: A Multicenter Narrative Medicine Project in Italy
por: Simonelli, Francesca, et al.
Publicado: (2021)

Evaluation for Retinal Therapy for RPE65 Variation Assessed in hiPSC Retinal Pigment Epithelial Cells
por: Nash, Benjamin M., et al.
Publicado: (2021)

Validation of a Vision-Guided Mobility Assessment for RPE65-Associated Retinal Dystrophy
por: Kumaran, Neruban, et al.
Publicado: (2020)

The interplay of environmental luminance and genetics in the retinal dystrophy induced by the dominant RPE65 mutation
por: Wu, Wenjing, et al.
Publicado: (2022)

Lentiviral mediated RPE65 gene transfer in healthy hiPSCs-derived retinal pigment epithelial cells markedly increased RPE65 mRNA, but modestly protein level
por: Udry, Florian, et al.
Publicado: (2020)

Systemic Injection of RPE65-Programmed Bone Marrow-Derived Cells Prevents Progression of Chronic Retinal Degeneration
por: Qi, Xiaoping, et al.
Publicado: (2017)

Cannot write session to /tmp/vufind_sessions/sess_nic8tuspm8v2egcr6fcvpnu6ie