Výsledky vyhledávání - "Jörn Magnus Schenk"

Akademický článek

A 15-Year Perspective of the Fabry Outcome Survey

Autor: Roberto Giugliani MD, PhD, Dau-Ming Niu MD, PhD, Uma Ramaswami MD, Michael West MD, Derralynn Hughes MD, Christoph Kampmann MD, PhD, Guillem Pintos-Morell MD, PhD, Kathleen Nicholls MD, Jörn-Magnus Schenk MD, PhD, Michael Beck MD

Publikováno v: Journal of Inborn Errors of Metabolism and Screening, Vol 4 (2016)

The Fabry Outcome Survey (FOS) is an international long-term observational registry sponsored by Shire for patients diagnosed with Fabry disease who are receiving or are candidates for therapy with agalsidase alfa (agalα). Established in 2001, FOS p

Externí odkaz: https://doaj.org/article/63f79614ee0b4238ae2229500163e13e

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Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report

Autor: Stephanie Westwood, Christoph Kampmann, Hartmann Wellhoefer, Andrey Gurevich, Roberto Giugliani, Jörn Magnus Schenk

Publikováno v: Repositório Institucional da UFRGS
Universidade Federal do Rio Grande do Sul (UFRGS)
instacron:UFRGS
Genetics and Molecular Biology v.41 n.4 2018
Genetics and Molecular Biology
Sociedade Brasileira de Genética (SBG)
instacron:SBG
Genetics and Molecular Biology, Volume: 41, Issue: 4, Pages: 790-793, Published: 11 OCT 2018
Genetics and Molecular Biology, Vol 41, Iss 4, Pp 790-793 (2018)
Genetics and Molecular Biology, Issue: ahead, Published: 11 OCT 2018

Anderson-Fabry disease (AFD) is a rare lysosomal storage disorder. Randomized controlled clinical trials (RCTs) are preferred as the highest category of evidence, but limited availability of robust evidence in rare diseases may necessitate the use of

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3b84ffead0aeca38119f43520b81d300

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