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Mucopolysaccharidosis IVA (Morquio A Syndrome)
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis IVA (Morquio A Syndrome) trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis IVA (Morquio A Syndrome) trials you may qualify forThis 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 purpose of this study is to better understand how heart and blood vessel problems develop in people with Mucopolysaccharidosis (MPS). The investigators are…
Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establi…
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.…
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple…
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…