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A Scan of Pleiotropic Immune Mediated Disease Genes Identifies Novel Determinants of Baseline FVIII Inhibitor Status in Hemophilia-A

2023-10-18

Abstract excerpt

<title>Abstract</title> <p>Hemophilia-A (HA) is caused by heterogeneous loss-of-function factor (F)VIII gene (<italic>F8</italic>)-mutations and deficiencies in plasma-FVIII-activity that impair intrinsic-pathway-mediated coagulation-amplification. The standard-of-care for severe-HA-patients is regular infusions of therapeutic-FVIII-proteins (tFVIIIs) but ~30% develop neutralizing-tFVIII-antibodies called “FVIII-...

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Literature Corpus work
eedfe30f-6710-5a46-8955-96a4576c0c05
DOI
10.21203/rs.3.rs-3371095/v1
Open publication

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