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

Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A

11 Apr 2024

Abstract excerpt

BACKGROUND: Valoctocogene roxaparvovec transfers a human factor (F)VIII coding sequence into hepatocytes of people with severe hemophilia A to provide bleeding protection. OBJECTIVES: To present 3-year efficacy and safety in the multicenter, open-label, single-arm, phase 3 GENEr8-1 trial. METHODS: GENEr8-1 enrolled 134 adult males with severe hemophilia A who were receiving FVIII prophylaxis. Efficacy endpoints...

Read the complete abstract on PubMed

Topics

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

Share to Complement system in diseases
Share to Hemophilia Treatment and Research
Share to Virus-based gene therapy research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 38614387
DOI: 10.1016/j.jtha.2024.04.001

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia APMID 38614387Read ↗

Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trialSemantic proximity Read ↗

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia BSemantic proximity Read ↗

Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta studySemantic proximity Read ↗

Roctavian gene therapy for hemophilia ASemantic proximity Read ↗

Etranacogene dezaparvovec for hemophilia B gene therapySemantic proximity Read ↗

Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trialSemantic proximity Read ↗

The Arrival of Gene Therapy for Patients with Hemophilia ASemantic proximity Read ↗

Gene therapy for hemophiliaSemantic proximity Read ↗

Gene-based therapies for hemophiliaSemantic proximity Read ↗

Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia BSemantic proximity Read ↗

Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia BSemantic proximity Read ↗

Liver Gene TherapySemantic proximity Read ↗

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