Hypophosphatemic rickets, autosomal recessive, 1
MONDO:0009430Any autosomal recessive hypophosphatemic rickets in which the cause of the disease is a mutation in the DMP1 gene.
Also known as: DMP1 autosomal recessive hypophosphatemic rickets, autosomal recessive hypophosphatemic rickets caused by mutation in DMP1, hypophosphatemic rickets, AR, hypophosphatemic rickets, autosomal recessive, 1, hypophosphatemic rickets, autosomal recessive, type 1, ARHR1, Arhr, hypophosphatemia, autosomal recessive
5 clinical trials for this condition and its sub-types, 5 tagged with Hypophosphatemic rickets, autosomal recessive, 1 itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →-
New hope for babies: drug targets rare genetic disorder that hardens arteries
Disease control OngoingThis study tests a new medicine called INZ-701 in babies up to 1 year old who have a rare genetic condition (ENPP1 deficiency) that causes severe hardening of the arteries and bone problems. The goal is to see if the drug can raise a key substance in the blood, improve survival, …
Phase 3 • Sponsor: Inozyme Pharma • Aim: Disease control
Last updated Aug 12, 2026 00:00 UTC
-
New hope for babies with rare calcification disease: first drug trial launches
Disease control OngoingThis study tests a new drug called INZ-701 in up to 16 infants (up to 1 year old) with rare genetic conditions (ENPP1 or ABCC6 deficiency) that cause dangerous calcium buildup in arteries and other problems. The main goal is to check the drug's safety and how the body handles it,…
Phase 1 • Sponsor: Inozyme Pharma • Aim: Disease control
Last updated Aug 12, 2026 00:00 UTC