Mutation in gene ATP7A, which encodes ATP-dependent copper efflux protein in the intestine → copper can be absorbed into de the mucosal cell, but cannot be transported into the bloodstream
X-linked recessive
Incidence 1/100.000
AKA Ehlers-Danlos type IX or kinky hair syndrome
Pathophysiolosy
Collagen pathway
Copper is co-factor for Lysil oxidase - makes collagen rigid (not elastic)
Oxida a hidroxilisina e faz cross-linking covalente
Clinical presentation
Cutaneous
Pigment dilution (rare reports)
Pale skin
Sparse kinky hypopigmented hair
Non-cutaneous
Arterial turtuosity and rupture
Cerebral degeneration
Osteoporosis
Anemia
Bladder diverticula
Labs
Ceruloplasmin is not a Cu transporter!! It oxidizes Fe2+ to Fe3+ and requires Cu to work