Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes

Fabry disease is a rare inherited condition that can lead to serious renal, cardiac and cerebrovascular events. Using pooled clinical trial data, we examined the efficacy of migalastat, an oral treatment, in a large group of patients. The rate of newly occurring Fabry-associated clinical events (FACES) with prolonged treatment was low, regardless of whether patients had previously received enzyme replacement therapy (ERT). FACES were more common in patients who had renal impairment at study entry. Findings suggest that migalastat is an effective long-term treatment option for patients with Fabry disease. Preserving kidney function and timely treatment initiation instead of a watch-and-wait monitoring strategy may improve patient outcomes. (By Prof. Derralynn Hughes, https://jmg.bmj.com/content/early/2022/12/21/jmg-2022-108669 )

eGFR, estimated glomerular filtration rate, was used to indicate renal impairment

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