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

Malte Lenders Peter 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.

Keyphrases

left ventricular
end stage renal disease
emergency department
newly diagnosed
primary care
heart failure
hypertrophic cardiomyopathy
coronary artery disease
single cell
combination therapy
atrial fibrillation
aortic stenosis
left atrial
smoking cessation