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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Principais autores:	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
פורמט:	Artigo
שפה:	Inglês
יצא לאור:	BMC 2025-03-01
סדרה:	Orphanet Journal of Rare Diseases
נושאים:	Enzyme replacement therapy Self-administration Agalsidase alfa Velaglucerase alfa Safety Fabry
גישה מקוונת:	https://doi.org/10.1186/s13023-024-03416-2
תגים:	הוספת תג אין תגיות, היה/י הראשונ/ה לתייג את הרשומה!