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Gaucher DiseaseRare disease

What this study is

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.

Study details

Sponsor
Duke University
Conditions
Gaucher Disease
Enrolling
200 people
Starts
2017-06-12
Est. completion
2028-01
Record
View on ClinicalTrials.gov ↗