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

Pub. Date : 2022 May 5

PMID : 35512362






1 Functional Relationships(s)
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1 AIMS: Fabry disease (FD) is an X-linked lysosomal storage disorder caused by a deficiency of the lysosomal enzyme alpha-galactosidase A (GLA/AGAL), resulting in the lysosomal accumulation of globotriaosylceramide (Gb3). globotriaosylceramide alpha 1,4-galactosyltransferase (P blood group) Homo sapiens