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Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, …
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford HealthNCT01793168
Natural History Study of Adult and Pediatric Patients With Hypophosphatasia
Hypophosphatasia (HPP) is a rare inherited metabolic disorder characterized by defective bone and teeth mineralization caused by mutations of the ALPL gene, wh…
Hypophosphatasia
Duke UniversityNCT02237625
A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia
In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will …
Hypophosphatasia
Alexion Pharmaceuticals, Inc.NCT05234567