Last updated on August 2015

Rare disease registries for certain lysosomal storage disorders, including, but not limited to, Fabry, Gaucher, MPS I, and Pompe diseases (each, a Registry)


Brief description of study

Rare disease registries for certain lysosomal storage disorders, including, but not limited to, Fabry, Gaucher, MPS I, and Pompe diseases (each, a Registry)

Detailed Study Description

The Rare Disease Registry Program (including but not limited to, Gaucher, Fabry, MPS I, and Pompe diseases) is a multi-center, international, longitudinal, observational program that tracks the natural history and outcomes of patients with these rare diseases.

Clinical Study Identifier: TX141086

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Recruitment Status: Open


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