ReviewAnnals of translational medicine2019
Wilson disease-treatment perspectives.
Review in Annals of translational medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers, 2 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
32 citing papers in PubMed, 2 syntheses or guidelines pooled it, 60 citations in OpenAlex.
- Early neurological deterioration in Wilson's disease: a systematic literature review and meta-analysis.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2023Pooled it
- Liver transplantation as a treatment for Wilson's disease with neurological presentation: a systematic literature review.Acta neurologica Belgica · 2022Pooled it
- Effect of tiomolibdate choline on copper balance in patients with Wilson disease: An open-label phase 2 trial.Hepatology communications · 2026Trial
- Social Support, Self-Management Behaviors, and Coping Styles of Patients With Wilson's Disease: A Quantitative Empirical Research.Brain and behavior · 2025Article
- Wilson disease: time frame for improvement of neurological symptomology may exceed a decade.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025Article
- Wilson's Disease in Oman: A National Cohort Study of Clinical Spectrum, Diagnostic Delay, and Long-Term Outcomes.Clinics and practice · 2025Article
- Hepatic progenitor cells reprogrammed from mouse fibroblasts repopulate hepatocytes in Wilson's disease mice.Stem cell research & therapy · 2025Article
- Copper-Induced Neurodegenerative Disorders and Therapeutic Potential of Curcumin-Loaded Nanoemulsion.Toxics · 2025Article
- The Role of Glia in Wilson's Disease: Clinical, Neuroimaging, Neuropathological and Molecular Perspectives.International journal of molecular sciences · 2024Review
- Characteristics of patients with Wilson disease in the United States: An insurance claims database study.World journal of hepatology · 2024Article
- Review
- Treatment patterns in a real-world cohort of patients with Wilson disease in the United States.Frontiers in gastroenterology (Lausanne, Switzerland) · 2024Article
- Effect of high-frequency repetitive transcranial magnetic stimulation on cognitive impairment in WD patients based on inverse probability weighting of propensity scores.Frontiers in neuroscience · 2024Article
- Exploring Copper's role in stroke: progress and treatment approaches.Frontiers in pharmacology · 2024Review
- D-Penicillamine-Induced Myasthenia Gravis-A Probable Complication of Wilson's Disease Treatment-A Case Report and Systematic Review of the Literature.Life (Basel, Switzerland) · 2023Article
- "Because it is a rare disease…it needs to be brought to attention that there are things out of the norm": a qualitative study of patient and physician experiences of Wilson disease diagnosis and management in the US.Orphanet journal of rare diseases · 2023Article
- Serum neurofilament light chain and initial severity of neurological disease predict the early neurological deterioration in Wilson's disease.Acta neurologica Belgica · 2023Article
- Blood Based Biomarkers of Central Nervous System Involvement in Wilson's Disease.Diagnostics (Basel, Switzerland) · 2023Review
- Analysis of risk factors for neurological symptoms in patients with purely hepatic Wilson's disease at diagnosis.BMC neurology · 2023Article
- Ferulic Acid Activates SIRT1-Mediated Ferroptosis Signaling Pathway to Improve Cognition Dysfunction in Wilson's Disease.Neuropsychiatric disease and treatment · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Wilson disease (WD) is a genetic disorder caused by pathological tissue copper accumulation with secondary damage of affected organs (mainly, but not limited to, the liver and brain). The main clinical symptoms of WD are, in concordance with the pathogenesis, hepatic and/or neuropsychiatric. Current treatment options for WD, based on drugs leading to negative copper body balance like chelators or zinc salts, were introduced more than 40 years ago and are generally effective in the majority of WD cases if used lifelong. However, especially in neurological patients, treatment may lead to neurological deterioration, which is often irreversible. Further, almost 50% of neurologically affected WD patients present with persistent neurological deficits despite the use of anti-copper treatment. In addition, up to 30% of patients treated with the widely used drug, d-penicillamine, present with adverse events related to treatment, which often leads to treatment discontinuation. Finally, almost 25% of WD patients do not adhere with anti-copper treatment, partially due to drug-related adverse events and complex treatment regimens (3 times daily, before meals, etc.). These limitations with current treatments have led to the search for other WD treatment possibilities. Currently, research is mainly focused on: (I) new agents with better safety profiles and less neurological deterioration properties compared with traditional chelators, e.g., tetrathiomolybdate salts or central nervous system-penetrable trientine, with the aim to provide more effective copper removal from brain tissue; (II) other non-chelating drugs that lead to removal of copper from cells [e.g., methanobactin (currently in preclinical studies)]; (III) cell and gene therapy. In this article, current research on future treatments for WD is reviewed.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.