Comprehensive Pharmacological Management of Wilson’s Disease: Mechanisms, Clinical Strategies, and Emerging Therapeutic Innovations

Wilson’s disease is a rare autosomal recessive disorder of copper metabolism characterized by excessive copper accumulation in the liver, brain, and other tissues. This paper provides an overview of the primary pharmacological agents used in its treatment, including penicillamine, trientine, tetrathiomolybdate, and zinc. Their mechanisms of action, therapeutic applications, and side-effect profiles are examined, emphasizing how each agent helps reduce copper overload. Additionally, brief information is given on novel therapies such as gene therapy and artificial intelligence applications. Furthermore, information about the structural and chemical properties of these compounds is provided, highlighting the molecular features that enable them to chelate copper or reduce its intestinal absorption. By integrating pathophysiological insights with chemical and mechanistic perspectives, this paper offers a comprehensive review of existing treatment strategies for Wilson’s disease and stresses the importance of careful, patient-specific management to optimize long-term outcomes.

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Journal
Sci
Published
2025-07-01
DOI
https://doi.org/10.3390/sci7030094
Citations
4
Primary Topic
Trace Elements in Health
Type
article
Field-Weighted Citation Impact
4.48

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article

Comprehensive Pharmacological Management of Wilson’s Disease: Mechanisms, Clinical Strategies, and Emerging Therapeutic Innovations

Ralf Weiskirchen
4 citations
Sci
Trace Elements in Health
4.48
article

Comprehensive Pharmacological Management of Wilson’s Disease: Mechanisms, Clinical Strategies, and Emerging Therapeutic Innovations

Ralf Weiskirchen
article en
4 citations

Abstract

Wilson’s disease is a rare autosomal recessive disorder of copper metabolism characterized by excessive copper accumulation in the liver, brain, and other tissues. This paper provides an overview of the primary pharmacological agents used in its treatment, including penicillamine, trientine, tetrathiomolybdate, and zinc. Their mechanisms of action, therapeutic applications, and side-effect profiles are examined, emphasizing how each agent helps reduce copper overload. Additionally, brief information is given on novel therapies such as gene therapy and artificial intelligence applications. Furthermore, information about the structural and chemical properties of these compounds is provided, highlighting the molecular features that enable them to chelate copper or reduce its intestinal absorption. By integrating pathophysiological insights with chemical and mechanistic perspectives, this paper offers a comprehensive review of existing treatment strategies for Wilson’s disease and stresses the importance of careful, patient-specific management to optimize long-term outcomes.

SciVol. 7(3)
Universitätsklinikum Aachen (DE), RWTH Aachen University (DE)
Deutsche Forschungsgemeinschaft, Deutsche Krebshilfe, RWTH Aachen University
Openalex Percentile: Top 6%
Trace Elements in Health
4.48
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Comprehensive Pharmacological Management of Wilson’s Disease: Mechanisms, Clinical Strategies, and Emerging Therapeutic Innovations — Ralf Weiskirchen · Sci (2025) | TGRS Research Map | TGRS