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Non-invasive Functional Assessment and Pathogenesis of Morquio A (NIFAMA)
Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establ…
Mucopolysaccharidosis IV Type AMorquio A SyndromeMPS IVA
Nemours Children's ClinicNCT05284006
Phase 1
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety an…
MPS IMPS IIMPS IVA+6 more
University of California, San FranciscoNCT04532047
Cardiovascular Structure and Function in Mucopolysaccharidosis Types I and IVA
The purpose of this study is to better understand how heart and blood vessel problems develop in people with Mucopolysaccharidosis (MPS). The investigators are…
MPS IMPS IV AMPS - Mucopolysaccharidosis+1 more
Children's Hospital of Orange CountyNCT07361536