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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First treatment tested for babies with deadly artery hardening disease
Disease control Recruiting nowThis study is testing a new enzyme replacement therapy called INZ-701 in infants with rare genetic disorders that cause dangerous calcium buildup in arteries and weak bones. The main goal is to check if the treatment is safe and well-tolerated in babies under one year old. Resear…
Matched conditions: ATP-BINDING CASSETTE SUBFAMILY C MEMBER 6 DEFICIENCY
Phase: PHASE1 • Sponsor: Inozyme Pharma • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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Global hunt for clues in devastating rare diseases affecting babies and adults
Knowledge-focused Recruiting nowThis study aims to learn more about two rare, inherited diseases that cause serious bone problems and blood vessel calcification, particularly in infants. Researchers will follow up to 1000 patients over time during their regular doctor visits, collecting health information and a…
Matched conditions: ATP-BINDING CASSETTE SUBFAMILY C MEMBER 6 DEFICIENCY
Sponsor: Inozyme Pharma • Aim: Knowledge-focused
Last updated Mar 03, 2026 14:07 UTC