Gaucherite stratified medicine project
Full Study Name
Predictive measures to stratify clinical outcomes in children and adults with Gaucher disease and responses to specific therapies
Cambridge University Hospitals NHS Foundation Trust
Local Principal Investigator
To develop a clinical stratification of Gaucher disease
- Confirmed biochemical diagnosis of Type 1 or Type 3 Gaucher disease
- Written Ethics Committee (EC) approved informed consent obtained from the patient, or patient’s parent or legal
- guardian and patient assent if appropriate.
- Male or Female Patients Aged ≥5 years
- Willing to comply with study schedule and procedures
- Unrelated comorbid
- condition limiting life expectancy to less than 6 months.
- Confirmed Type 2 Neurological Gaucher Disease.
- Patient or if applicable, parent or legal guardian is unable to comprehend, sign and date the EC approved informed
- consent form and patient assent as appropriate.
- If determined unsuitable for the study by the investigator.
Birmingham, Great Ormond Street, Salford, UCLH, Manchester, Royal Free
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