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Osteonecrosis in Gaucher Disease in the era of multiple therapies: biomarker set for risk stratification from a tertiary referral center

2023-03-27

Abstract excerpt

<h4>BACKGROUND</h4> A salutary effect of treatments for Gaucher disease (GD) has been reduction in the incidence of avascular osteonecrosis (AVN). However, there are reports of AVN in patients receiving enzyme replacement therapy (ERT), and it is not known whether it is related to individual treatments, GBA genotypes, phenotypes, biomarkers of residual disease activity or anti-drug antibodies. <h4>OBJECTIVE</h4...

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Literature Corpus work
946b6980-dc2e-537e-b8bc-6d3032985786
DOI
10.1101/2023.03.21.23287476
Open publication

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Osteonecrosis in Gaucher Disease in the era of multiple therapies: biomarker set for risk stratification from a tertiary referral centerDOI 10.1101/2023.03.21.23287476
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