The Patient Experience of Gene Therapy for Hemophilia: Qualitative Interviews with Trial Patients

BACKGROUND: The phase ½ hemophilia B clinical trial (AMT-060) demonstrated stable endogenous FIX levels after 3.5 years (mean FIX activity between 5.1% and 7.5%) with continued reductions in annualized bleeds to near zero with the higher dose, and a 78–96% reduction by year in exogenous FIX use. OBJ...

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Detalles Bibliográficos
Autores principales: Miesbach, Wolfgang, Klamroth, Robert
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove 2020
Materias:
Case Series
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7184858/
https://www.ncbi.nlm.nih.gov/pubmed/32368018
http://dx.doi.org/10.2147/PPA.S239810

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7184858/
https://www.ncbi.nlm.nih.gov/pubmed/32368018
http://dx.doi.org/10.2147/PPA.S239810

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