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Bis‐choline Tetrathiomolybdate as Old Drug in a New Design for Wilson’s Disease: Good for Brain and Liver?
Author(s) -
Stremmel Wolfgang
Publication year - 2019
Publication title -
hepatology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 5.488
H-Index - 361
eISSN - 1527-3350
pISSN - 0270-9139
DOI - 10.1002/hep.30130
Subject(s) - choline , drug , brain disease , wilson's disease , liver disease , medicine , pharmacology , disease , neuroscience , psychology
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.