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Treatment of Fabry Disease management with migalastat-outcome from a prospective 24 months observational multicenter study (FAMOUS).

Malte LendersPeter NordbeckChristine KurschatMaria EveslageNesrin KarabulJessica KaufeldJulia B HennermannMonica PattenMarkus CybullaJonas MüntzeNurcan ÜçeylerDan LiuAnibh M DasClaudia SommerChristian PogodaStefanie ReiermannThomas DuningJens GaedekeKatharina von CosselDaniela BlaschkeStefan-Martin BrandW Alexander MannChristoph KampmannNicole MuscholSima Canaan-KühlEva Brand
Published in: European heart journal. Cardiovascular pharmacotherapy (2022)
Treatment with migalastat was generally safe and resulted in most patients in an amelioration of left ventricular mass. However, due to the heterogeneity of FD phenotypes, it is advisable that the treating physician monitors the clinical response regularly.
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