Article

Real Life Event-Based Outcomes of Patients With Gaucher Disease Receiving ERT

2023-11-08

Abstract excerpt

<h4>Purpose: </h4> Gaucher Disease (GD) is a lysosomal storage disease. Mutations in GBA1 gene cause glucocerebrosidase enzyme deficiency that leads accumulation of its substrates. Clinical presentations of GD are cytopenia, hepatosplenomegaly, bone involvement and neurological impairment. Besides; abdominal pain, painful bone crises, bleeding, growth retardation, delayed puberty, pulmonary involvement, fatigue ar...

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Literature Corpus work: 396087ad-b989-59e9-9c80-a4327a8069c8
DOI: 10.21203/rs.3.rs-3557812/v1

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