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

~Estimated

~$10 – $750

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

~Estimated

~$10 – $5,625

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

~Estimated

~$10 – $750

Children's Hospital of Orange CountyNCT07361536