| Literature DB >> 32640076 |
Aleš Linhart1, Dominique P Germain2, Iacopo Olivotto3, Mohammed M Akhtar4, Aris Anastasakis5, Derralynn Hughes6, Mehdi Namdar7, Maurizio Pieroni8, Albert Hagège9,10,11, Franco Cecchi3,12, Juan R Gimeno13, Giuseppe Limongelli14, Perry Elliott4.
Abstract
Fabry disease (FD) is an X-linked lysosomal storage disorder caused by pathogenic variants in the α-galactosidase A (GLA) gene that leads to reduced or undetectable α-galactosidase A enzyme activity and progressive accumulation of globotriaosylceramide and its deacylated form globotriaosylsphingosine in cells throughout the body. FD can be multisystemic with neurological, renal, cutaneous and cardiac involvement or be limited to the heart. Cardiac involvement is characterized by progressive cardiac hypertrophy, fibrosis, arrhythmias, heart failure and sudden cardiac death. The cardiac management of FD requires specific measures including enzyme replacement therapy or small pharmacological chaperones in patients carrying amenable pathogenic GLA gene variants and more general management of cardiac symptoms and complications. In this paper, we summarize current knowledge of FD-related heart disease and expert consensus recommendations for its management.Entities:
Keywords: Cardiomyopathy; Enzyme replacement therapy; Fabry disease; GLA gene
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Year: 2020 PMID: 32640076 DOI: 10.1002/ejhf.1960
Source DB: PubMed Journal: Eur J Heart Fail ISSN: 1388-9842 Impact factor: 15.534