This trial is evaluating whether INZ-701 will improve 1 primary outcome and 5 secondary outcomes in patients with Pseudoxanthoma Elasticum. Measurement will happen over the course of 32 days (Dose Evaluation Period).
This trial requires 9 total participants across 1 different treatment group
This trial involves a single treatment. INZ-701 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.
"This is a rare disease (estimated prevalence of 1 in 1,000,000). The cause is still not known, but it is likely due to a combination of genetic, environmental, and other pathogenic alterations to tissue. PSE is one of the most important causes of blindness and vision loss in the Western world." - Anonymous Online Contributor
"This is the first study to show potential therapy combining tazarotene and isotretinoin for the treatment of PXE. Tazarotene and isotretinoin demonstrated promising effects in improving clinical, histological, and genetic parameters of PXE and provided a promising therapeutic option for future trials." - Anonymous Online Contributor
"This is the first study to demonstrate that pseudoxanthoma elasticum patients have a higher prevalence of cardiovascular disease, but no association with non-cardiovascular disease, compared with healthy control subjects." - Anonymous Online Contributor
"The present case of a patient with PE presenting with systemic symptoms warrants consideration of a differential diagnosis. A history of previous dental trauma in patients with PE or a history of dental pulp neoplasm suggests a possible dental or paracapsular or accessory radicular cyst in the area. The diagnosis of PE in this case was confirmed with the help of ultrasound examination of the subcutaneous tissues and in vivo confocal laser Doppler imaging of the oral cavity. The present situation lends further credence to the use and effectiveness of an integrated approach to the treatment of PE." - Anonymous Online Contributor
"In a recent study, findings demonstrated that a family history of PXE and pseudoxanthoma elasticum-like skin features were associated with clinical features of PXE. In a recent study, findings also showed that pseudoxanthoma elasticum can be used as a clinical marker to identify mutations of FBXL3." - Anonymous Online Contributor
"PXE is a rare genetic condition in the general population and is inherited in an X-linked recessive fashion. PXE is thought to be inherited by parents from the same X-chromosome. Inheritance in an X-linked recessive fashion means that both parents must be carriers of the defective gene. For this reason, the disease is inherited in an X-linked dominant fashion, wherein the affected child is likely to inherit an X-chromosome containing the defective gene from either parent. More than 60% of PXE cases appear to be sporadic and may occur at an older age; women and men are affected equally." - Anonymous Online Contributor
"There is no cure for PXE but treatments can increase life expectancy and reduce symptoms. Common treatments for PXE include skin care, lifestyle modifications, counselling, orthoses, and medication." - Anonymous Online Contributor
"There are no specific records-based estimates of PXE in the US. PXE-affected individuals are at all ages and are usually asymptomatic until significant organ or systemic diseases develop due to the severity of the disease. There appears to be a higher incidence of PXE in individuals of non-Caucasian descent. The cause of PXE is hereditary. No known risk factors have been shown to increase the predisposition to PXE. In fact, family and genetic counseling are recommended for all patients with PXE. It is not clear when PXE is diagnosed in early childhood, but the first recorded case of PXE was seen in a baby girl in 1860 in France." - Anonymous Online Contributor
"The new topical agent I-701 was found to be more effective than a placebo in slowing down the progression of PXE in this patient population." - Anonymous Online Contributor
"Clinical trials are the most effective tool for the treatment of PXE. Patients with severe disease should consider clinical trials; patients with mild disease may opt for self-monitoring." - Anonymous Online Contributor
"It is common that pseudoxanthoma elasticum presents as palpable papules and plaques. In most cases, the cause is not precisely known, but it may be caused by various systemic disorders, the immune system or genetics. The primary cause has to be identified and treated to prevent the complication of eschara." - Anonymous Online Contributor