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The article reported by Weiss et al. describes in a phase II trial showing the efficacy of bis-choline tetrathiomolybdate (TTM) in 28 patients with Wilson disease (WD) (1). After 24 weeks of treatment, 71% of patients met the primary endpoint of normalized non-ceruloplasmin-bound copper (NCC) (57%) or a ≥ 25% reduction of NCC (14%). This was accompanied by an improvement of neurological status. A reversible increase of transaminases and gamma-glutamyltransferase occurred in 39% of patients who received at least 30 mg/day. In 25% of patients, 11 serious adverse events were reported of which 7 were unlikely to be related to the study drug. This article is protected by copyright. All rights reserved.

Bis‐choline Tetrathiomolybdate as Old Drug in a New Design for Wilson’s Disease: Good for Brain and Liver?