ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE1 DEFICIENCY
Clinical trials for ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE1 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: ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE1 DEFICIENCY
Phase: PHASE1 • Sponsor: Inozyme Pharma • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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Hope for babies with Ultra-Rare fatal disease: experimental drug enters final testing phase
Disease control Recruiting nowThis study is testing an investigational drug called INZ-701 in infants with a severe, ultra-rare genetic disorder called ENPP1 deficiency. The main goals are to see if the drug improves survival and prevents dangerous calcium buildup in the heart and blood vessels. About 12 infa…
Matched conditions: ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE1 DEFICIENCY
Phase: PHASE3 • Sponsor: Inozyme Pharma • Aim: Disease control
Last updated Mar 23, 2026 15:16 UTC