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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...
| Autores principales: | , , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2021
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| Materias: | |
| 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 |