Multiple Sulfatase Deficiency Disease

2

Review clinical trials related to Multiple Sulfatase Deficiency Disease. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

MPS (RaDiCo Cohort) (RaDiCo-MPS)

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.

Participants needed: 1,000
Trial details
Biological sex: AllType: ObservationalSponsor: Institut National de la Santé Et de la Recherche Médicale, FranceUpdated: Feb 11, 2026Locations: 23
Eligibility criteria

Confirmed diagnosis of MPS based on clinically relevant enzyme deficiency, with... [+1]

Status: Recruiting

Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

Participants needed: 1,500
Trial details
Biological sex: AllType: ObservationalSponsor: University of PittsburghUpdated: Feb 9, 2026Locations: 1
Eligibility criteria

Any patient with a genetic neurodegenerative disorder

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