RecruitingNCT03190837

A Long-term Follow-up Study of Gaucher Disease


Sponsor

Duke University

Enrollment

200 participants

Start Date

Jun 12, 2017

Study Type

OBSERVATIONAL

Conditions

Summary

The study aims are to: a) identify the long-term natural history of Gaucher disease, b) evaluate long-term treatment efficacy of enzyme replacement therapy (ERT) and substrate reduction therapy (SRT), and c) identify potential long-term complications of this disorder. These aims will be accomplished through long-term record review of individuals with all three types of Gaucher disease.


Eligibility

Inclusion Criteria1

  • Diagnosis of Gaucher Disease

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Locations(1)

Duke University Medical Center

Durham, North Carolina, United States

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NCT03190837


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