Intra-monocyte Pharmacokinetics of Imiglucerase Supports a Possible Personalized Management of Gaucher Disease Type 1.
Juliette BergerMarie ViganBruno PereiraThu Thuy NguyenRoseline FroissartNadia BelmatougFlorence DalbièsAgathe MasseauChristian RoseChristine SerratriceYves-Marie PersIvan BertchanskyFabrice CamouMonia BengherbiaCéline BourgneCatherine CaillaudMagali PettazzoniAmina BerrahalJérôme StirnemannFrance MentréMarc G BergerPublished in: Clinical pharmacokinetics (2020)
This study provides novel pharmacokinetic data that support current imiglucerase administration regimens and suggests the existence of a glucocerebrosidase activity threshold related to Gaucher disease type 1 aggressiveness. These findings can potentially improve Gaucher disease type 1 management algorithms and clinical decision making.