1 recruiting. 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Recruiting 1 |
Follow Mps VII: new trials by email Literature Review of Mps VII trials Research Report on Mps VII
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
Gaucher Disease (1) · Mucopolysaccharidosis IV (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Sudden Infant Death (1) · Wolman Disease (1)
Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Carbohydrate Metabolism, Inborn Errors (1) · Central Nervous System Diseases (1) · Cholesterol Ester Storage Disease (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Connective Tissue Diseases (1) · Death (1) · Death, Sudden (1) · Genetic Diseases, Inborn (1) · Infant Death (1) · Infant, Newborn, Diseases (1) · Lipid Metabolism Disorders (1) · Lipid Metabolism, Inborn Errors (1)
GAA protein, human (1) · GALNS protein, human (1) · Iduronidase (1) · Sebelipase alfa (1) · galsulfase (1) · idursulfase (1) · vestronidase alfa (1)
University of California, San Francisco (1)
United States (1)
PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 28374935, PMID 28657663, PMID 30337566, PMID 32102934, PMID 37955580, PMID 39891377, PMID 9918480