Condition: Gaucher Disease · Sponsor: Duke University
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.
This description comes directly from the study's public registry record.
Janet Blount · 919-681-7962 · janet.blount@duke.edu
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| Duke University Medical Center | Durham, North Carolina, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT03190837