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

~Estimated

~$10 – $1,125

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

~Estimated

~$10 – $750

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

~Estimated

~$10 – $8,750

Alexion Pharmaceuticals, Inc.NCT05234567