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MPS VII clinical trials

View clinical trials related to MPS VII.

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NCT ID: NCT04532047 Recruiting - Wolman Disease Clinical Trials

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

IUERT
Start date: July 1, 2021
Phase: Phase 1
Study type: Interventional

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

NCT ID: NCT03604835 Recruiting - Clinical trials for Mucopolysaccharidosis VII

Mucopolysaccharidosis VII Disease Monitoring Program

Start date: January 29, 2018
Phase:
Study type: Observational

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.