Research.Patient.info
Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency research hub
2 indexed studies · 2 currently recruiting
- recruiting
PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency
This study aims to understand rare conditions called ENPP1 Deficiency and ABCC6 Deficiency. Researchers will gather information from medical records and routine check-ups to learn more about how these conditions develop and affect people over time. It's an observational study, meaning there's no new treatment or intervention.
United States · Canada · Germany - 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