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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...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Dove
2020
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| 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 |