Predictive Measures to Stratify Clinical Outcomes in Children and Adults With Gaucher Disease and Responses to Specific Therapies
Status: Recruiting
Location: See all (8) locations...
Intervention Type: Other
Study Type: Observational
SUMMARY
The purpose of this research is to review data already collected and to collect new data from adults and children in England with Gaucher Disease to determine clinical factors which predict severity and response to therapy of Gaucher disease especially in the areas of bone, cancer and brain conditions.
Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
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⁃ Each patient must meet all of the following criteria to be enrolled in this study:
• Confirmed biochemical diagnosis of Type I, Type II or Type III 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, no age limitation
• Willing and able to comply with study schedule and procedures
• Deceased patients for whom the EC determines that patient data can be collected without a new consent from the patient