Article

Eye movement biomarkers allow for the definition of phenotypes in Gaucher Disease

2020-10-28

Abstract excerpt

<title>Abstract</title> <p>Background Neurological forms of Gaucher disease, the inherited disorder of β-Glucosylceramidase caused by bi-allelic variants in GBA1, is a progressive disorder which lacks a disease-modifying therapy. Systemic manifestations of disease are effectively treated with Enzyme Replacement Therapy (ERT), however, molecules which cross the blood-brain barrier are still under investigation. C...

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Literature Corpus work: da2d7e70-2c21-54fd-88e6-7a12b7544be5
DOI: 10.21203/rs.3.rs-37355/v2

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