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editorial
. 2018 Jul 7;8(3):153–157. doi: 10.15171/bi.2018.17

Table 1. Approved enzyme replacement therapies available for the lysosomal storage disorders .

LSDs Deficient enzyme Inheritance FDA approved ERT and Brand name
MPS I (Hurler syn.)
MPS II (Hunter syn.)
MPS IV A (Morquio A syn.)
MPS VI (Marateaux-Lamy syn.)
α-L-iduronidase
Iduronate sulfatase
N-acetylgalactosamine 6-sulfatase
N-acetylgalactosamine 4-sulfatase
Autosomal
X-linked
Autosomal
Autosomal
Laronidase (Aldurazyme™)/ 2003-FDA, EMA
Idursulfase (Elaprase™)/ 2006-FDA; 2007-EMA
Elosulfase Alfa (Vimzim™)/ 2014-FDA
Galsulfase (Naglazyme™)/ 2005-FDA; 2006-EMA
Fabry disease α-galactosidase X-linked Agalsidase α (Fabrazyme™)/ 2001-EMA
Agalsidase β (Replagal™)/ 2003-FDA, EMA
Pompe diseas α-glucosidase Autosomal Aglucosidase (Myozyme™)/ 2006-FDA, EMA
Aglucosidase (Lumizyme™)/ 2010-FDA
Gaucher disease β -glucocerebrosidase Autosomal Aglucerase (Ceredase™)/ 1991-FDA
Imiglucerase (Cerezyme™)/ 1994-FDA; 1997-EMA
Velaglucerase (VPRIV™)/ 2010-FDA, EMA
Taliglucerase (Elelyso™)/ 2012-FDA
Lysosomal acid lipase deficiency Lysosomal acid lipase Autosomal Sebelipase α (Kanuma™)/ 2015-FDA,EMA

MPS: mucopolysaccharidosis; FDA: U.S. Food and Drug Administration; EMA: European Medical Agency.1,5,7