Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys

Abstract Background Fabry disease and Gaucher disease are rare genetic disorders characterized by defective degradation of glycosphingolipids caused by enzymatic deficiencies in α–galactosidase A and β–glucocerebrosidase, respectively, and often require life-long treatment. Treatment options for the...

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發表在:	Orphanet Journal of Rare Diseases
Main Authors:	Shoshana Revel-Vilk, Uma Ramaswami, Guillem Pintos-Morell, Derralynn Hughes, Kathy Nicholls, Ricardo Reisin, Roberto Giugliani, Ozlem Goker-Alpan, Majdolen Istaiti, Aidan Gill, Maurizio Scarpa, Jaco Botha
格式:	Article
語言:	英语
出版:	BMC 2025-03-01
主題:	Enzyme replacement therapy Self-administration Agalsidase alfa Velaglucerase alfa Safety Fabry
在線閱讀:	https://doi.org/10.1186/s13023-024-03416-2