Pseudoxanthoma Elasticum
Pseudoxanthoma Elasticum (PXE) is a rare genetic disorder that affects the skin, eyes, and cardiovascular system, leading to symptoms like yellowish bumps on the skin, vision problems, and arterial calcification.
We are studying the long-term safety of INZ-701 in patients with ENPP1 and ABCC6 deficiencies. This trial will help us understand how the drug works over time and its effects on certain blood markers.
Health conditions and diseases that the clinical trial is designed to study and treat.
Pseudoxanthoma Elasticum (PXE) is a rare genetic disorder that affects the skin, eyes, and cardiovascular system, leading to symptoms like yellowish bumps on the skin, vision problems, and arterial calcification.
Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency is a rare genetic condition that can lead to generalized arterial calcification and other complications, often presenting in infancy.
Generalized Arterial Calcification of Infancy (GACI) is a rare genetic disorder characterized by the abnormal buildup of calcium in the arteries, which can lead to serious cardiovascular issues in infants.
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.