A Long-term Follow-up Study of Gaucher Disease
Recruiting
Observational Study
Gaucher Disease
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 200 (estimated)
- Sponsor
- Duke University · Other
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Answer a few quick questions to see if you may meet the eligibility requirements.
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Diagnosis of Gaucher Disease
Exclusion Criteria:
* Not meeting Inclusion criteria
Contacts