A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
| Status: | Completed |
|---|---|
| Conditions: | Other Indications, Metabolic |
| Therapuetic Areas: | Pharmacology / Toxicology, Other |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 2/4/2017 |
| Start Date: | July 2013 |
| End Date: | January 2015 |
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.
clinical characteristics and course of disease progression of MPS IIIB.
Inclusion Criteria:
1. The deceased patient was diagnosed with MPS IIIB as determined by either of the
following:
1. Documented deficiency of NAGLU enzyme activity.
2. Documented functionally-relevant mutations in both alleles of the NAGLU gene.
2. Patient's parent or legal guardian provides informed consent (unless the requirement
for informed consent is waived by local regulations).
3. 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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