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Mucopolysaccharidosis Type VI
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis Type VI trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis Type VI trials may be worth asking aboutNorth America
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This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dise…
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and…
The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collect…
This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI.…
Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared t…