Actively Recruiting
A Long-term Follow-up Study of Gaucher Disease
Led by Duke University · Updated on 2025-09-22
200
Participants Needed
1
Research Sites
498 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Long-term Follow-up Study of Gaucher Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Gaucher Disease
You will not qualify if you...
- Not meeting Inclusion criteria
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
Research Team
J
Janet Blount
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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