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Hypophosphatemic Rickets
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Hypophosphatemic Rickets trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Hypophosphatemic Rickets trials you may qualify forX-linked hypophosphataemia (XLH) is a rare, hereditary condition. The genetic defect leads to low blood phosphate levels and vitamin D suppression. Phosphate is…
FGF23 is the cornerstone of phosphate / calcium / vitamin D metabolism: it is synthesized mainly by osteocytes and acts as a phosphaturizing agent, inhibitor of…
The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 De…
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase/phosphodiesterase1(ENPP1) Deficiency and the i…
A first-in-human study of KK8123 in adults with X-linked hypophosphatemia.
Children and adults with XLH recruited will be treated with calcitriol alone (without phosphate supplementation) for one year, during which the calcitriol dose…
Background: Generalized Arterial Calcification of Infancy (GACI) is a very rare disorder. It can be fatal before birth or by age 6 months. Anumber of people wi…
The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of buro…