RecruitingNCT06036693
MPS (RaDiCo Cohort) (RaDiCo-MPS)
Mucopolysaccharidosis Patients in France in the Era of Specific Therapeutics
Sponsor
Institut National de la Santé Et de la Recherche Médicale, France
Enrollment
1,000 participants
Start Date
Dec 20, 2017
Study Type
OBSERVATIONAL
Conditions
Summary
The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collection of extensive phenotypic data from French MPS patients.
Eligibility
Inclusion Criteria2
- Confirmed diagnosis of MPS based on clinically relevant enzyme deficiency, with abnormally elevated GAG urinary excretion and/or identification of pathogenic mutations.
- Signed informed consent or parents/guardian non-opposition for deceased patients (minor or protected major)
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Locations(23)
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NCT06036693
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