Valoctocogene roxaparvovec transfers a human factor (F)VIII coding sequence into hepatocytes of people with severe hemophilia A to provide bleeding protection.

Ask AI

Helpful

Bookmark

View Full Paper

Ask AI

Helpful

Bookmark

View Full Paper

Cite This Study

Madan et al. (Fri,) studied this question.

synapsesocial.com/papers/68e6f5edb6db64358766fec7 https://doi.org/https://doi.org/10.1016/j.jtha.2024.04.001

Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1Long‐term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post‐treatment2024 · 47 citations
2Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A2022 · 406 citations
3Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors2001 · 315 citations
4Joint disease in haemophilia: Pathophysiology, pain and imaging2018 · 174 citations
5Mildly Elevated Liver Transaminase Levels: Causes and Evaluation.2017 · 128 citations

Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1Long‐term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post‐treatment2024 · 47 citations
2Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A2022 · 406 citations
3Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors2001 · 315 citations
4Joint disease in haemophilia: Pathophysiology, pain and imaging2018 · 174 citations
5Mildly Elevated Liver Transaminase Levels: Causes and Evaluation.2017 · 128 citations