Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Real-world outcomes of voretigene neparvovec treatment in pediatric patients with RPE65-associated Leber congenital amaurosis.

Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie - 1 May 2022

Deng Callie, Zhao Peter Y, Branham Kari, Schlegel Dana, Fahim Abigail T, Jayasundera Thiran K, Khan Naheed, Besirli Cagri G

Abstract excerpt

PURPOSE: To investigate real-world safety and efficacy of voretigene neparvovec gene therapy administration in pediatric patients with biallelic RPE65 disease-causing variants. METHODS: A retrospective study of 27 eyes of 14 patients with RPE65-associated Leber congenital amaurosis examined postoperative complications and longitudinal changes in photoreceptor function following treatment with subretinal injection...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Child
Share to cis-trans-Isomerases

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Leber Congenital Amaurosis

Share to Mutation

Share to Retina

Share to Tomography, Optical Coherence

Share to Visual Acuity

Source

Open in PubMed Open full text

Identifiers

PMID: 35001204
DOI: 10.1007/s00417-021-05508-2
PMCID: PMC9010358

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Real-world outcomes of voretigene neparvovec treatment in pediatric patients with RPE65-associated Leber congenital amaurosis.PMID 35001204Read ↗

Single Center Experience with Voretigene Neparvovec Gene Augmentation Therapy in RPE65 Mutation-Associated Inherited Retinal Degeneration in a Clinical Setting.Semantic proximity Read ↗

Voretigene neparvovec for inherited retinal dystrophy due to RPE65 mutations: a scoping review of eligibility and treatment challenges from clinical trials to real practice.Semantic proximity Read ↗

Gene therapy outcomes in young patients with RPE65-retinal degeneration.Semantic proximity Read ↗

Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical EvidenceSemantic proximity Read ↗

Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.Semantic proximity Read ↗

Chorioretinal atrophy following voretigene neparvovec despite the presence of fundus autofluorescence.Semantic proximity Read ↗

Voretigene Neparvovec and Gene Therapy for Leber’s Congenital Amaurosis: Review of Evidence to DateSemantic proximity Read ↗

Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy.Semantic proximity Read ↗

Voretigene Neparvovec for the Treatment of RPE65-associated Retinal Dystrophy: Consensus and Recommendations from the Korea RPE65-IRD Consensus Paper Committee.Semantic proximity Read ↗

Long-term effect of gene therapy on Leber's congenital amaurosis.Semantic proximity Read ↗

12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with RPE65 -mediated inherited retinal dystrophySemantic proximity Read ↗

Visual function and retinal changes after voretigene neparvovec treatment in children with biallelic RPE65-related inherited retinal dystrophySemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.