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Pseudoxanthoma elasticum (PXE) is an autosomal-recessive multisystem disease characterized by elastic fiber calcification in the skin, the cardiovascular system, and eyes. PXE is caused by biallelic mutations in the ABCC6 gene on chromosome 16, resulting in low inorganic pyrophosphate plasma levels. Typical ocular manifestations result from calcification of Bruch's membrane and include peau d'orange, angioid streaks, and comet-tail lesions. Advances in multimodal imaging have significantly improved the detection of these features. Ocular complications in PXE include the development of secondary neovascularization, which can be treated with anti-vascular endothelial growth factor (VEGF) injections. In cases of suspected PXE, genetic testing and genetic counseling should be performed for diagnostic confirmation. Early referral to a specialized center is essential. Interdisciplinary care and management of complications, particularly in collaboration with angiologists, are crucial for treating this multisystem disorder. Although no approved causal therapy for PXE currently exists to date, accurate and early diagnosis using phenotypic characteristics is critical. Early identification of other affected family members and timely treatment of secondary complications allow for better disease management and offer patients the opportunity to participate in ongoing clinical trials. Promising therapeutic options are currently emerging and may significantly improve management and prognosis in the future.
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http://dx.doi.org/10.1007/s00347-025-02261-3 | DOI Listing |
Cureus
August 2025
Ophthalmology, Burjeel Medical City, Abu Dhabi, ARE.
We present a case of a 23-year-old female with characteristic skin papules and angioid streaks characteristic of pseudoxanthoma elasticum (PXE), an autosomal recessive disorder of elastic fiber mineralization. Genomic sequencing revealed a heterozygous variant in the ABCC6 gene. Despite the absence of biallelic mutations, the clinical phenotype aligns with PXE.
View Article and Find Full Text PDFBiochem Biophys Rep
September 2025
Guangdong Cardiovascular Institute, Guangdong Provincial People's Hospital, Guangdong Academy of Medical Sciences, Southern Medical University, Guangzhou, Guangdong, 510100, China.
Pseudoxanthoma elasticum (PXE), caused by pathogenic variants in , is characterized by pathological ectopic calcification with poorly understood mechanisms and no effective therapies. To address this, we developed the first zebrafish model of human PXE by introducing the pathogenic point mutation ( , F2 generation) using the highly efficient zhyA3A-CBE5 cytosine base editor. Three mutant types (Type1-Type3, T1-T3) stratified by calcification severity, exhibited reduced levels of the calcification inhibitors vitamin K1 (VK1) and carboxylated matrix Gla protein (cMGP), which were inversely correlated with the severity of calcification.
View Article and Find Full Text PDFAnn Dermatol Venereol
August 2025
PXE National Reference Center, MAGEC Nord, University Hospital of Angers, Angers, France; University of Angers, MITOVASC Laboratory, UMR CNRS 6015, INSERM U1083, Angers, France. Electronic address:
Ann Dermatol Venereol
August 2025
Allergology and Photobiology Department Université Grenoble Alpes, Dermatology,- CHU Grenoble Alpes, Grenoble, France; Institute for Advanced Biosciences, Université Grenoble Alpes, INSERM U1209, Grenoble, France.
Invest Ophthalmol Vis Sci
August 2025
Department of Ophthalmology, University of Bonn, Bonn, Germany.
Purpose: To assess acute IOP changes after anti-VEGF injections in patients with Pseudoxanthoma elasticum (PXE) compared to other retinal diseases.
Methods: Twenty eyes of patients with PXE (mean age 63.4 ± 6.