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Article

Patient and caregiver burden of neuronopathic Gaucher disease: The GARDIAN Registry experience

2026-05-25

Abstract excerpt

<title>Abstract</title> <p>Background Neuronopathic Gaucher disease (nGD) is a rare, progressive condition with substantial neurological and systemic burden and limited treatment options. Patient-reported outcome data are sparse and often rely on agnostic instruments that may not capture the full disease-specific impact. The Gaucher Registry for Development, Innovation and Analysis of Neuronopathic disease (GARD...

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Literature Corpus work
8d0598db-67cc-54ad-921e-6667d345cc58
DOI
10.21203/rs.3.rs-9198845/v1
Open publication

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Patient and caregiver burden of neuronopathic Gaucher disease: The GARDIAN Registry experienceDOI 10.21203/rs.3.rs-9198845/v1
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