The objectives of this study are to describe the clinical and biochemical characteristics and course of disease progression in participants with Mucopolysaccharidosis type IIIB (MPS IIIB)
Study Type
OBSERVATIONAL
Enrollment
30
Unnamed facility
Minneapolis, Minnesota, United States
Unnamed facility
Pittsburgh, Pennsylvania, United States
Unnamed facility
Porto Alegre, Brazil
Unnamed facility
Monza, Italy
Collection And Analysis Of Clinical Characteristics Of MPS IIIB
Component 1 involved an evaluation of the clinical characteristics of MPS IIIB in participants based on a retrospective chart review to collect information on demographics, clinical history, diagnostic tests, treatments, clinical chemistry and hematology test results, physical examination findings, anthropometric data, radiology results, and supportive interventions performed over a period of up to 6 weeks.
Time frame: Baseline to Week 43
Longitudinal Analysis Of The Course Of Disease Progression In Participants With MPS IIIB
Component 2 involved a longitudinal evaluation of the course of disease progression in a subset of participants considered to be at risk of rapid disease progression, who, after completing Component 1, were to be prospectively followed for a period of at least 1 year (Longitudinal Follow-Up) and up to 3 years total (Extended Follow-Up).
Time frame: Baseline to Week 43
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Unnamed facility
Coimbra, Portugal
Unnamed facility
Barcelona, Spain
Unnamed facility
Birmingham, United Kingdom