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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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Gardado en:
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
Formato: Artigo
Idioma:Inglês
Publicado: BMC 2025-03-01
Series:Orphanet Journal of Rare Diseases
Assuntos:
Enzyme replacement therapy
Self-administration
Agalsidase alfa
Velaglucerase alfa
Safety
Fabry
Acceso en liña:https://doi.org/10.1186/s13023-024-03416-2
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