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

Longitudinal Study of Neurodegenerative Disorders

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 understandi…

MLDKrabbe DiseaseALD+24 more

~Estimated

~$10 – $6,250

University of PittsburghNCT03333200