Pseudoxanthoma Elasticum research hub
3 indexed studies · 1 currently recruiting
- recruitingNA
Purinergic Compounds in Pseudoxanthoma Elasticum
This study looks into a rare genetic condition called Pseudoxanthoma Elasticum (PXE), which causes body tissues to stiffen. Researchers are exploring how certain natural body chemicals, like ATP and pyrophosphate, might be linked to this condition. The aim is to understand PXE better and find new ways to treat it.
France - active not 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 recruitingPHASE2
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
This study looks at the long-term safety of a treatment called INZ-701 for people with rare conditions like ENPP1 Deficiency or ABCC6 Deficiency. It's for patients who have already tried INZ-701 in a previous study and wish to continue.
United States · France · Germany