Pearls & Oy-sters: Challenges and Controversies in Wilson Disease.

Ruiz-Lopez, Marta; Moreno Estébanez, Ana; Tijero, Beatriz; Fernandez, Tamara; Rebollo-Perez, Alba; Gabilondo, Iñigo; Lopez-Osle, Nuria; Ceberio-Hualde, Leticia et al. · Neurology · 2022

case_series · Level IV

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Abstract

Wilson disease (WD) is a genetic disorder of copper metabolism caused by variants in the <i>ATP7B</i> gene, which are inherited in an autosomal recessive pattern. Despite all the advances made on pathogenesis, cellular biology, and genetics, to date, WD remains a diagnostic and therapeutic challenge. With this series of cases, we aim to illustrate the main challenges that clinicians may encounter when dealing with patients with WD: the difficulties with clinical diagnosis, the therapeutic management of WD and the indication for advanced therapies, management during pregnancy, and genotype-phenotype correlations.

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