Research.Patient.info
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency research hub
2 indexed studies · 1 currently recruiting
- recruitingPHASE1
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
The ENERGY Study is looking into a new medicine called INZ-701 for babies under one year old with two rare genetic conditions: ENPP1 Deficiency or ABCC6 Deficiency. The main goal is to check how safe the medicine is and if babies can tolerate it well.
United States · Spain · United Kingdom - active not recruitingPHASE3
The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency
The ENERGY 3 Study is looking at a new treatment called INZ-701 for children aged 1-12 with a rare genetic condition called ENPP1 Deficiency. This study wants to see how well INZ-701 works and if it's safe compared to usual care.
United States · Australia · Canada