ArticleMolecular genetics and metabolism reports2026
Effectiveness and tolerability of migalastat in adult Fabry disease: A single regional centre experience.
Article in Molecular genetics and metabolism reports, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Fabry disease (FD) is an X-linked lysosomal storage disorder caused by deficient α-galactosidase A (α-Gal) activity, leading to progressive renal, cardiac, and cerebrovascular involvement. Migalastat, an oral pharmacological chaperone, is indicated for patients with amenable Methods: Eighty-seven adults with FD (55 males, 32 females) carrying Results: Variants were predominantly late-onset (67%), most commonly c.644 A > G (p.Asn215Ser)/p.N215S; 14% were classical and 19% unclassified. Plasma globotriaosylsphingosine (lyso-Gb3) declined in both groups (TN: 5.1 (3.7-7.9) to 2.2 (1.3-3.6) ng/mL; TS: 7.2 (3.8-13.0) to 3.3 (1.4-6.7) ng/mL). Renal function remained above clinically relevant thresholds in most patients, with a modest reduction in the proportion maintaining eGFR >90 mL/min/1.73 m Conclusion: Migalastat was well tolerated and maintained stable renal and cardiac parameters with plasma lyso-Gb3 improvement. However, a subset of patients showed progression or intolerance, underscoring that variant amenability alone may not predict clinical benefit.
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