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A phase II, multicenter, open-label trial to evaluate the safety and efficacy of ISU303 (Agalsidase beta) in patients with Fabry disease

Fabry disease (FD) is caused by a deficiency in the activity of the lysosomal enzyme, α-galactosidase A (α-Gal A), which leads to globotriaosylceramide (Gb3) deposition in multiple tissues. The current management of FD is enzyme replacement therapy (ERT). We report on the efficacy and safety of a ne...

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Detalles Bibliográficos
Autores principales:	Hwang, Soojin, Lee, Beom Hee, Kim, Woo-Shik, Kim, Dae-Seong, Cheon, Chong Kun, Lee, Chang Hwa, Choi, Yunha, Choi, Jin-Ho, Kim, Ja Hye, Yoo, Han-Wook
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Lippincott Williams & Wilkins 2022
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9478233/ https://www.ncbi.nlm.nih.gov/pubmed/36123934 http://dx.doi.org/10.1097/MD.0000000000030345

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