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Mucopolysaccharidosis Type IVA
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis Type IVA trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis Type IVA 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 understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understandin…
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.…
Mucopolysaccharidosis IVA (MPS IVA) (or Morquio A disease) is a rare recessive autosomal lysosomal storage disorder caused by deficiency of N-acetylgalactosamin…
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that prov…