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- W4311341125 abstract "Gaucher Disease (GD) is a rare inherited metabolic disorder. Type 2 and Type 3 are neuronopathic and often result in infant death or progressive neurological deterioration. Current drug therapies do not cross the blood brain barrier and thus do not treat neuronopathic GD (nGD). GARDIAN data will generate real-world evidence on the natural history and impact of disease and will inform clinical trial design and healthcare decision making. The objective is to describe the development of a patient registry specific to nGD." @default.
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- W4311341125 date "2022-12-01" @default.
- W4311341125 modified "2023-09-27" @default.
- W4311341125 title "SA73 Addressing Unmet Needs of Patients With Neuronopathic Gaucher Disease Type 2 and Type 3: Creation of the GARDIAN Patient Registry" @default.
- W4311341125 doi "https://doi.org/10.1016/j.jval.2022.09.2466" @default.
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