Long-term follow-up for Gaucher disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to understand the long-term natural history of Gaucher disease by evaluating the efficacy of enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). It involves systematic monitoring of health records from patients diagnosed with all three types of Gaucher disease. Health information will be collected from medical records at Duke University and from treating physicians outside the institution. The principal investigator, Dr. Kishnani, is a recognized expert in the field and will oversee the annual monitoring of patient health status without altering their standard care.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Diagnosis of Gaucher Disease

Exclusion Criteria:

* Not meeting Inclusion criteria

Where it's running

• Duke University Medical Center
  Durham, North Carolina, United States Recruiting

Study contacts

• Principal investigator: Priya Kishnani, MD — Duke University
• Study coordinator: Janet Blount
• Email: janet.blount@duke.edu
• Phone: 919-681-7962

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Long-term observation of rare genetic brain disorders

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.