ATP-BINDING CASSETTE SUBFAMILY C MEMBER 6 DEFICIENCY
Clinical trials for ATP-BINDING CASSETTE SUBFAMILY C MEMBER 6 DEFICIENCY explained in plain language.
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New hope for babies with rare calcification disease: experimental drug enters human testing
Disease control Recruiting nowThis study tests an experimental drug called INZ-701 in 16 infants (up to 1 year old) with rare genetic conditions (ENPP1 or ABCC6 deficiency) that cause dangerous calcium buildup in blood vessels and other tissues. The main goal is to check the drug's safety and how the body pro…
Matched conditions: ATP-BINDING CASSETTE SUBFAMILY C MEMBER 6 DEFICIENCY
Phase: PHASE1 • Sponsor: Inozyme Pharma • Aim: Disease control
Last updated May 15, 2026 11:55 UTC
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New registry aims to map rare genetic diseases that harden arteries in babies
Knowledge-focused Recruiting nowThis study is a patient registry that will follow up to 1,000 people with ENPP1 deficiency or infantile-onset ABCC6 deficiency. Researchers will collect information from routine doctor visits, including genetic, imaging, and quality-of-life data, to better understand how these ra…
Matched conditions: ATP-BINDING CASSETTE SUBFAMILY C MEMBER 6 DEFICIENCY
Sponsor: Inozyme Pharma • Aim: Knowledge-focused
Last updated May 17, 2026 06:51 UTC