Recruiting PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) NCT04532047 Conditions Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +39 more Locations University of California — San Francisco, California, United States View Trial Details
Completed Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome NCT01961518 Conditions Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +14 more Locations Greenwood Genetic Center — Greenville, South Carolina, United States View Trial Details
Completed Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 NCT01387854 Conditions Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more Locations Children's Hospital and Research Center — Oakland, California, United States Women's and Children's hospital — North Adelaide, Australia Hospital de Clinicas de Porto Alegre — Porto Alegre, Brazil Hôpital Femme Mère Enfant — Lyon, France View more locations Show fewer locations Loading locations... View Trial Details
Active, Not Recruiting Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI NCT05845749 Conditions Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more Locations UCSF Benioff Children's Hospital Oakland — Oakland, California, United States View Trial Details
