Abstract
Advances in Treatment of Wilson Disease
Highlights
Wilson disease (WD) was first described by S.A.K
It was soon realized that dimercaprol was not a tenable longterm treatment option as it was associated with considerable adverse effects, rapid drug tolerance, and waning clinical benefits
We focus on definitive medical treatments that directly address the root issue of excessive copper deposition in WD
Summary
Wilson disease (WD) was first described by S.A.K. Wilson, in 1912, as a new syndrome of familial lentiform degeneration and liver cirrhosis, which was invariably fatal.[1]. The first copper chelator used was intramuscular dimercaprol, which increased urinary copper excretion and resulted in remarkable clinical improvement.[3,4,5] it was soon realized that dimercaprol was not a tenable longterm treatment option as it was associated with considerable adverse effects, rapid drug tolerance, and waning clinical benefits. It was penicillamine, introduced by John Walshe in 1955, that changed the prognosis for WD, making survival as well as dramatic and sustained clinical recovery possible. For each drug used to treat WD, we surveyed its development, indication for use, dosing, efficacy, and adverse effects
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