Review clinical trials related to Mucopolysaccharidosis VII. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.
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Not listed
The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.
Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic o... [+2]
Concurrent participation in other pharmaceutical company-sponsored interventiona...
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.
Patients aged 0-64 with a diagnosis of a lysosomal storage disease [+1]
There are no current exclusion criteria
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.
Confirmed diagnosis of MPS based on clinically relevant enzyme deficiency, with... [+1]