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Early identification of Wilson disease — a rare autosomal recessive disorder of copper transport — allows prompt treatment, which can improve outcomes. Treatments for patients with symptomatic disease include chelating agents such as D-penicillamine or trientene. However, adverse effects can limit the use of these agents. An alternate treatment, zinc therapy (which inhibits copper uptake in the gastrointestinal tract) is well tolerated, but its use has been limited to patients with asymptomatic disease.
To evaluate the efficacy of zinc monotherapy in symptomatic Wilson disease, investigators at a single center assessed 17 patients (mean age, 18) who presented with hepatic features only (7 patients), neurological features only (5 patients), o…