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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...

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Vydáno v:Patient Prefer Adherence
Hlavní autoři: Miesbach, Wolfgang, Klamroth, Robert
Médium: Artigo
Jazyk:Inglês
Vydáno: Dove 2020
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7184858/
https://ncbi.nlm.nih.gov/pubmed/32368018
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/PPA.S239810
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