Clinical Efficacy and Real-World Effectiveness of Fabry Disease Treatments: A Systematic Literature Review. [PDF]
J Clin MedJovanovic A +9 more
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Impact of ER stress and the unfolded protein response on Fabry disease. [PDF]
EBioMedicineLenders M, Rudolph E, Brand E.
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Hypertrophic Cardiomyopathy and Phenocopies: New Therapies for Old Diseases-Current Evidence and Future Perspectives. [PDF]
J Clin MedAlfarano M +13 more
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Therapeutic Alternatives to Recombinant Biologics: Mechanistic Framework, Clinical Evidence, and Selection Guidance. [PDF]
Drug Des Devel TherNiazi SK.
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WCN24-2159 REAL-WORLD EVIDENCE REGARDING RENAL EFFECT OF MIGALASTAT
Kidney International ReportsSebastián Jaurretche +5 more
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Biochemical Amenability in Fabry Patients Under Chaperone Therapy-How and When to Test? [PDF]
BioDrugsLenders M, Menke ER, Brand E.
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Modeling Fabry nephropathy: insights from experimental systems towards precision nephrology. [PDF]
Dis Model MechElsaid H +4 more
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Effects of Current Therapies on Disease Progression in Fabry Disease: A Narrative Review for Better Patient Management in Clinical Practice. [PDF]
Adv TherMignani R +6 more
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Pathogenicity of novel GLA gene missense mutations in Fabry disease and the therapeutic impact of migalastat. [PDF]
J Adv ResGuo W, Ji P, Li Y, Zhang Y, Bi J, Xie Y.
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Autophagy-Lysosomal Dysfunction as a Converging Mechanism of Cardiomyopathy in Lysosomal Storage Disorders: From Pathobiology to Targeted Therapy. [PDF]
Int J Mol SciLee CL +8 more
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