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Learn about Hemophilia A topics

Safety and Efficacy Profile

Publication

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Safety and Efficacy Profile

Estimated long-term durability of valoctocogene roxaparvovec treatment in male patients with severe hemophilia a: An extrapolation of clinical data

Comparative effectiveness of valoctocogene roxaparvovec and efanesoctocog alfa in the treatment of severe hemophilia a: A matching-adjusted indirect comparison of bleeding frequency

Plain language summary of 4-year results of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia a

A plain language summary on indirectly comparing bleeding after valoctocogene roxaparvovec gene therapy to bleeding with emicizumab prophylaxis

Safety and efficacy of valoctocogene roxaparvovec in participants with active or prior FVIII inhibitors: Results from a phase 1/2 trial

Impact of gene therapy on the hemophilia-free mind: Outcomes before and after valoctocogene roxaparvovec in GENEr8-1 trial participants

Final analysis of the phase 1/2 trial of valoctocogene roxaparvovec for severe hemophilia A

Efficacy and safety of valoctocogene roxaparvovec for severe hemophilia A 5 years after gene transfer in GENEr8-1

Valoctocogene roxaparvovec estimated long-term durability of treatment effect: An extrapolation of the most recent clinical data

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

Valoctocogene roxaparvovec estimated long-term durability of treatment effect: An extrapolation of the most recent clinical data

Final analysis of the phase 1/2 trial of valoctocogene roxaparvovec for severe hemophilia A

Valoctocogene roxaparvovec estimated long-term durability of treatment effect: An extrapolation of the most recent clinical data

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Bleeding rate, FVIII utilization, corticosteroid use, quality of life and safety 4 years after gene transfer with valoctocogene roxaparvovec in a Brazilian cohort: Results from GENEr8-1

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

How long do the effects of valoctocogene roxaparvovec last? A plain language summary of a model estimating how long patients with hemophilia A may experience benefit

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Plain language summary of 4-year efficacy and safety data in the GENEr8-1 trial of valoctocogene roxaparvovec, a gene therapy for severe hemophilia A

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Efficacy, safety and quality of life three years after gene transfer with valoctocogene roxaparvovec in a Brazilian cohort

Health-related quality of life outcomes 4 years after treatment with valoctocogene roxaparvovec

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

Seven-year follow-up of valoctocogene roxaparvovec gene therapy for hemophilia A

Results from the Taiwanese cohort in GENEr8-1: bleeding rates, factor VIII usage, quality of life and safety in severe hemophilia A patients after gene therapy

Health-related quality of life improvements following valoctocogene roxaparvovec gene therapy in people without bleeds or target joints at baseline: a post hoc analysis from GENEr8-1

Safety and efficacy of valoctocogene roxaparvovec gene transfer for severe hemophilia A: an update from 4 years after treatment

Bleeding Outcomes in Participants with Factor VIII Activity <5 IU/dL Post-Gene Transfer in GENEr8-1

Safety and efficacy of valoctocogene roxaparvovec with prophylactic corticosteroids: 1-year GENEr8-3 results

Safety and efficacy of valoctocogene roxaparvovec in participants with active and prior FVIII inhibitors: Preliminary results from GENEr8-INH, a phase 1/2 study

Health-related quality of life and long-term joint damage in people with severe haemophilia A in Brazil

Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A

Bleeding, FVIII activity, and safety 3 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

Bleeding Outcomes in Participants With Factor VIII Activity <5 IU/dL Post-Gene Transfer in GENEr8-1

Endogenous FVIII activity and procedure-related FVIII use and bleeding: Post hoc analysis of GENEr8-1

Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE

Gene therapy in hemophilia A: The impact of valoctocogene roxaparvovec on patient outcomes – initial results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 trial

Effects of valoctocogene roxaparvovec gene therapy in people with severe hemophilia A after 3 years

Bleeding, FVIII activity, and safety 3 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

Gene Therapy in Hemophilia A: the Impact of Valoctocogene Roxaparvovec on Patient Outcomes – Initial Results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 Trial

Valoctocogene Roxaparvovec Safety

Musculoskeletal health after gene therapy for Hemophilia A: a three-year follow-up study

Efficacy and safety of valoctocogene roxaparvovec gene transfer for severe hemophilia A: Results from the GENEr8 1 three year analysis

Endogenous FVIII activity and procedure-related FVIII use and bleeding: Post hoc analysis of GENEr8-1

Comparative effectiveness of valoctocogene roxaparvovec and prophylactic factor VIII replacement estimated through propensity scoring

Blood Biodistribution and Vector Shedding of Valoctocogene Roxaparvovec in People with Severe Hemophilia A Results from the Phase 3 GENEr8-1 Trial

Hemostatic results for up to 6 years following treatment with valoctocogene roxaparvovec, an AAV5 hFVIII SQ gene therapy for severe hemophilia A

Relationship between transgene produced FVIII and bleeding rates 2 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

Interim 52-Week Analysis of Immunogenicity to the Vector Capsid and Transgene-Expressed Human FVIII in GENEr8-1, a Phase 3 Clinical Study of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A

Use of immunosuppressives in patients with hemophilia receiving gene therapy: Evidence generation using a mixed-methods approach

Health-Related Quality of Life Over 2 Years Following Valoctocogene Roxaparvovec Adeno-Associated Virus Gene Transfer For Severe Hemophilia A: Results From GENEr8-1

Interim 52-Week Analysis of Immunogenicity to the Vector Capsid and Transgene-Expressed Human FVIII in GENEr8-1, a Phase 3 Clinical Study of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A

The Effect of Prophylactic Corticosteroid Treatment on Adeno-Associated Virus-Mediated Gene Therapy and Potential Mechanisms of Action

Efficacy and Safety of Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: Results from the GENEr8-1 year two analysis

Valoctocogene Roxaparvovec Gene Transfer in Participants with HIV

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Haemophilia A in the Phase 3 Trial GENEr8-1

Endogenous FVIII activity and procedure-related FVIII use and bleeding: Post hoc analysis of GENEr8-1

Efficacy and safety of valoctocogene roxaparvovec adeno-associated virus gene transfer for severe hemophilia A: Results from the Phase 3 GENEr8-1 Trial

Hemostatic response is maintained for up to 5 years following treatment with valoctocogene roxaparvovec, an AAV5-hFVIII-SQ gene therapy for severe hemophilia A

LIVER BIOPSIES – MOLECULAR ANALYSES Analysis of Liver Biopsies Following AAV5-HFVIII-SQ Administration Revealed Inter-Individual Variability in Transgene mRNA and Protein Production

Patient Reported Outcomes

Impact of gene therapy on the hemophilia-free mind: Outcomes before and after valoctocogene roxaparvovec in GENEr8-1 trial participants

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Comparative EQ-5D utilities for valoctocogene roxaparvovec gene therapy and emicizumab in people with severe hemophilia A using a matching-adjusted indirect comparison

Clinical and humanistic burden of people with severe haemophilia A treated with prophylaxis in Europe: a longitudinal analysis from the CHESS studies

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Bleeding rate, FVIII utilization, corticosteroid use, quality of life and safety 4 years after gene transfer with valoctocogene roxaparvovec in a Brazilian cohort: Results from GENEr8-1

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec