Zobrazeno 1 - 7
of 7
pro vyhledávání: '"migalastat 5"'
Publikováno v:
International Journal of Molecular Sciences
Volume 21
Issue 19
International Journal of Molecular Sciences, Vol 21, Iss 7397, p 7397 (2020)
Volume 21
Issue 19
International Journal of Molecular Sciences, Vol 21, Iss 7397, p 7397 (2020)
Fabry disease, an X-linked recessive lysosomal disease, results from mutations in the GLA gene encoding lysosomal &alpha
galactosidase A (&alpha
Gal A). Due to these mutations, there is accumulation of globotriaosylceramide (GL-3) in plasma
galactosidase A (&alpha
Gal A). Due to these mutations, there is accumulation of globotriaosylceramide (GL-3) in plasma
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::89cc5486335a2185a8066231a0006adc
Akademický článek
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Autor:
Narita, Ichiei, Ohashi, Toya, Sakai, Norio, Hamazaki, Takashi, Skuban, Nina, Castelli, Jeffrey P., Lagast, Hjalmar, Barth, Jay A.
Publikováno v:
Clinical & Experimental Nephrology; Feb2020, Vol. 24 Issue 2, p157-166, 10p
Autor:
Gatterer, Constantin1 (AUTHOR) constantin.gatterer@meduniwien.ac.at, Beitzke, Dietrich2 (AUTHOR), Graf, Senta1 (AUTHOR), Lenz, Max1 (AUTHOR), Sunder-Plassmann, Gere3 (AUTHOR), Mann, Christopher1 (AUTHOR), Ponleitner, Markus4 (AUTHOR), Manka, Robert5 (AUTHOR), Fritschi, Daniel6 (AUTHOR), Krayenbuehl, Pierre-Alexandre7 (AUTHOR), Kamm, Philipp8 (AUTHOR), Dormond, Olivier9 (AUTHOR), Barbey, Frédéric9 (AUTHOR), Monney, Pierre10 (AUTHOR) pierre.monney@chuv.ch, Nowak, Albina7,11 (AUTHOR) albina.nowak@usz.ch
Publikováno v:
Life (2075-1729). May2023, Vol. 13 Issue 5, p1213. 11p.
Autor:
Wu, Yi Shuan1 (AUTHOR), Khanna, Richie2 (AUTHOR), Schmith, Virginia1 (AUTHOR), Lun, Yi2 (AUTHOR), Shen, Jin‐Song2 (AUTHOR), Garcia, Anadina2 (AUTHOR), Dungan, Leo2 (AUTHOR), Perry, Anthony2 (AUTHOR), Martin, Lukas2 (AUTHOR), Tsai, Pai‐Chi2 (AUTHOR), Hamler, Rick2 (AUTHOR), Das, Anibh M.3 (AUTHOR), Schiffmann, Raphael4 (AUTHOR), Johnson, Franklin K.2 (AUTHOR) fjohnson@amicusrx.com
Publikováno v:
Clinical Pharmacology in Drug Development. Sep2021, Vol. 10 Issue 9, p1075-1088. 14p.
Autor:
Rusu, Elena-Emanuela, Zilisteanu, Diana-Silvia, Ciobotaru, Lucia-Mihaela, Gherghiceanu, Mihaela, Procop, Alexandru, Jurcut, Ruxandra-Oana, Dulamea, Adriana Octaviana, Sorohan, Bogdan Marian
Publikováno v:
Biomedicines; Jul2022, Vol. 10 Issue 7, pN.PAG-N.PAG, 17p
Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study
Autor:
Toya Ohashi, Norio Sakai, Jay A. Barth, Takashi Hamazaki, Nina Skuban, Hjalmar Lagast, Ichiei Narita, Jeffrey P. Castelli
Publikováno v:
Clinical and Experimental Nephrology
Background Fabry disease is a progressive X-linked lysosomal disorder. In this subgroup analysis of the global phase III ATTRACT study, the efficacy and safety of oral migalastat, a pharmacologic chaperone, were investigated in Japanese patients with
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::30f29edb46d4bb5f3ae3518f71b80dd7
https://doi.org/10.1007/s10157-019-01810-w
https://doi.org/10.1007/s10157-019-01810-w