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Mucopolysaccharidosis (MPS)
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis (MPS) trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis (MPS) trials may be worth asking aboutNorth America
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Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersens…
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establi…
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dise…
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), desi…
This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an i…