Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A

INTRODUCTION: Valoctocogene roxaparvovec is an investigational AAV5‐based factor VIII (FVIII) gene therapy that has demonstrated sustained clinical benefit in people with severe haemophilia A. AIM: To report safety, tolerability, efficacy, and quality of life (QOL) among participants who received va...

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Detalles Bibliográficos
Autores principales: Pasi, K John, Laffan, Michael, Rangarajan, Savita, Robinson, Tara M, Mitchell, Nina, Lester, Will, Symington, Emily, Madan, Bella, Yang, Xinqun, Kim, Benjamin, Pierce, Glenn F, Wong, Wing Yen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9291073/
https://www.ncbi.nlm.nih.gov/pubmed/34378280
http://dx.doi.org/10.1111/hae.14391

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9291073/
https://www.ncbi.nlm.nih.gov/pubmed/34378280
http://dx.doi.org/10.1111/hae.14391

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