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The objective is to perform a retrospective chart review to generate data to evaluate the clinical characteristics and course of disease progression of MPS IIIB.
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| Measure | Description | Time Frame |
|---|---|---|
| Evaluation of disease presentation and the course of disease progression including the end stage of the disease in patients with MPS IIIB, assessed from medical history data obtained by retrospective chart review of deceased patients. | Data obtained by retrospective chart review will include medical history data including patient demographics, disease manifestation onset, clinical history, diagnostic tests and treatments for MPS IIIB, supportive interventions/medications, clinical chemistry and haematology results, anthropometric data, radiology results, and autopsy findings. | N/A as this is a retrospective chart review of deceased patients |
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Inclusion Criteria:
The deceased patient was diagnosed with MPS IIIB as determined by either of the following:
Patient's parent or legal guardian provides informed consent (unless the requirement for informed consent is waived by local regulations).
The availability of pre-defined information in the patient's clinical chart:
Exclusion Criteria:
There are no exclusion criteria for this study.
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30 deceased patients with MPS IIIB
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pittsburgh | Pennsylvania | United States | ||||
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| ID | Term |
|---|---|
| D009084 | Mucopolysaccharidosis III |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| Porte Alegre |
| Brazil |
| Amsterdam | Netherlands |
| Barcelona | Spain |
| Birmingham | United Kingdom |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |