Disease Directory Pseudoxanthoma Elasticum
Connective Tissue

Pseudoxanthoma Elasticum

Also known as: PXE, Gronblad-Strandberg syndrome, ABCC6 deficiency

Prevalence

1 in 25,000–100,000

Onset

Childhood to early adulthood (skin), adulthood (vascular/ocular complications)

Type

Genetic — autosomal recessive

Gene

ABCC6

About Pseudoxanthoma Elasticum

Pseudoxanthoma elasticum is a systemic metabolic disorder caused by loss-of-function variants in ABCC6, a hepatic ABC transporter involved in purine nucleotide export and systemic mineralisation regulation. Progressive calcification and fragmentation of elastic fibres affects the skin, Bruch's membrane in the eye, and the arterial wall, leading to characteristic yellowish skin papules, vision-threatening choroidal neovascularisation, and premature cardiovascular disease. The condition is clinically heterogeneous with highly variable age of onset and severity of complications.

Common Clinical Features

Yellow xanthoma-like papules on the lateral neck and flexural skin creases Lax, redundant skin with loss of elasticity Angioid streaks on fundoscopy (breaks in Bruch's membrane) Choroidal neovascularisation and progressive central vision loss Premature peripheral arterial disease and claudication Gastrointestinal haemorrhage from calcified mucosal vessels Accelerated coronary artery disease in young adults

Clinical Trial Eligibility Tips

What to know before applying to Pseudoxanthoma Elasticum trials.

Ophthalmology records documenting angioid streaks and any history of choroidal neovascularisation are critical screening documents — obtain fluorescein angiography or OCT-A reports.

Molecular confirmation of biallelic ABCC6 pathogenic variants is required for most interventional trials; research and clinical panels targeting ABCC6 are widely available.

Cardiovascular risk factor documentation (lipids, ABI, coronary imaging if applicable) is commonly required as trials often stratify by vascular burden.

Patient Resources

Patient Organization

PXE International

Visit website ↗

Natural History Registry

PXE International Patient Registry

Join registry ↗

Orphanet

European reference resource for rare diseases (ORPHA:758)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

Search NORD ↗

Find recruiting Pseudoxanthoma Elasticum trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Pseudoxanthoma Elasticum. Updated daily.

Related Rare Diseases