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.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Evaluation of the clinical data of MPS like growth for each system
Timeframe: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like signs for each system
Timeframe: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like symptoms for each system
Timeframe: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like complications for each system
Timeframe: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like psychomotor milestones
Timeframe: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like cognitive evolution
Timeframe: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like handicap using scales adapted to multivisceral disease for all types of MPS
Timeframe: Through study completion, an average of 5 years
Evaluation of the clinical data of MPS like handicap using scales adapted to cognitive and neurologic disease for the types I, II, III VII
Timeframe: Through study completion, an average of 5 years
Evaluation of the radiological data of MPS like standard bone radiographs
Timeframe: Through study completion, an average of 5 years
Evaluation of the radiological data of MPS like abdominal echography
Timeframe: Through study completion, an average of 5 years
Evaluation of the radiological data of MPS like echocardiography
Timeframe: Through study completion, an average of 5 years
Evaluation of the radiological data of MPS like cerebral and medullar tomodensitometry
Timeframe: Through study completion, an average of 5 years
Evaluation of the radiological data of MPS like magnetic resonance imaging
Timeframe: Through study completion, an average of 5 years
Evaluation of the electrophysiological data of MPS like EMG
Timeframe: Through study completion, an average of 5 years
Evaluation of the electrophysiological data of MPS like EEG
Timeframe: Through study completion, an average of 5 years
Evaluation of the electrophysiological data of MPS like ERG
Timeframe: Through study completion, an average of 5 years
Evaluation of the biochemical data of MPS like urinary GAG before specific treatment
Timeframe: Through study completion, an average of 5 years
Evaluation of the biochemical data of MPS like urinary GAG during specific treatment
Timeframe: Through study completion, an average of 5 years
Evaluation of the biochemical data of MPS like enzyme activities before specific treatment
Timeframe: Through study completion, an average of 5 years
Evaluation of the biochemical data of MPS like enzyme activities during specific treatment
Timeframe: Through study completion, an average of 5 years
Evaluation of the biochemical data of MPS like specific antibodies
Timeframe: Through study completion, an average of 5 years
Evaluation of the molecular data of MPS
Timeframe: Through study completion, an average of 5 years