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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
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
University of PittsburghNCT03333200