ArticleJournal of clinical medicine2024
Overcoming Resistance in Anderson-Fabry Disease: Current Therapeutic Challenges and Future Perspectives.
Article in Journal of clinical medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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.
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Who cites it
9 citing papers in PubMed.
- Fabry Disease: An Updated Perspective and Review of Treatment and Therapies.Advances in therapy · 2026Review
- Is Fabry disease more prevalent than we think? Understanding the critical role of family screening can make all the difference.International urology and nephrology · 2026Article
- Review
- Longitudinal Adipokine and Lipid Profiles in Fabry Disease.Journal of clinical medicine · 2026Article
- Revisiting the Genetics of Hypertrophic Cardiomyopathy: From Sarcomeres to Polygenic Modulation and Clinical Translation.Journal of clinical medicine · 2026Review
- Unmasking the Apex: Multimodality Imaging for the Evaluation of Left Ventricular Apical Obliteration.Diagnostics (Basel, Switzerland) · 2026Review
- The Evolving Role of Cardiac Imaging in Hypertrophic Cardiomyopathy: Diagnosis, Prognosis, and Clinical Practice.Biomedicines · 2025Review
- Article
- Integrative Approaches in the Management of Hypertrophic Cardiomyopathy: A Comprehensive Review of Current Therapeutic Modalities.Biomedicines · 2025Review
Corrections and comments
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Authors and funding
13 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Anderson-Fabry disease (AFD) remains a therapeutic challenge despite advances in early diagnosis and the availability of enzyme replacement therapies (ERTs). While early initiation of therapy can mitigate disease progression, resistance mechanisms-such as the development of anti-drug antibodies-limit the efficacy of current treatments, particularly in patients with severe genetic variants. Chaperone therapy provides a targeted option for a subset of patients, yet significant gaps remain in treating those with complete enzyme deficiency. This perspective article explores the existing therapeutic landscape and reflects on emerging treatments, such as mRNA and gene therapies, which hold promise for overcoming the resistance mechanisms. By addressing the limitations of current pharmacological options and considering future innovations, this article aims to outline the path forward for more effective and personalized treatment strategies in Anderson-Fabry disease.
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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.