MPS (RaDiCo Cohort) (RaDiCo-MPS)

ConditionsMucopolysaccharidosis I Mucopolysaccharidosis II Mucopolysaccharidosis III Mucopolysaccharidosis IV Mucopolysaccharidosis VI Mucopolysaccharidosis VII Mucopolysaccharidosis IX Multiple Sulfatase Deficiency Disease

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

AgeNot listed

SponsorInstitut National de la Santé Et de la Recherche Médicale, France

About this trial

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 criteria

Qualifiers

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)

Disqualifiers

None

Trial design

Treatments tested in this trial

Not listed

Trial groups

No trial groups listed

Locations23

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Sponsors and collaborators

Institut National de la Santé Et de la Recherche Médicale, France

Lead sponsor

Source ClinicalTrials.gov