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Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency research hub

1 indexed studies · 1 currently recruiting

recruitingTherapeutic confirmatory (Phase III)
The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
The ENERGY 2 study is testing a new medicine called INZ-701 for babies with ENPP1 deficiency. This rare condition can cause harmful calcium build-up in blood vessels and heart problems. Researchers want to see if INZ-701 is safe, protects the heart, and helps babies grow well over one year.
Hungary · France · Sweden