Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT)

Gabor E. Linthorst; Dominique P. Germain; Carla E. M. Hollak; Derralynn Hughes; Arndt Rolfs; Christoph Wanner; Atul Mehta

doi:10.1016/j.ymgme.2010.11.155

Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT)

Gabor E. Linthorst
, Dominique P. Germain
, Carla E. M. Hollak
, Derralynn Hughes
, Arndt Rolfs
, Christoph Wanner
, Atul Mehta

Research output: Contribution to journal › Article › Academic › peer-review

Abstract

The shortage of enzyme for treatment of Fabry disease has caused anxiety among patients, physicians and governments. Following a request from the European Medicines Agency, consensus was reached on the temporary prioritization of patients for treatment based on disease severity and potential reversibility. Advice on follow-up of patients was agreed upon. This consensus is proposed to support the temporary guidelines issued throughout the period of ERT shortage, which will most likely last until April 2011

Original language	English
Pages (from-to)	99-102
Journal	Molecular genetics and metabolism
Volume	102
Issue number	1
DOIs	https://doi.org/10.1016/j.ymgme.2010.11.155
Publication status	Published - 2011

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title = "Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT)",

abstract = "The shortage of enzyme for treatment of Fabry disease has caused anxiety among patients, physicians and governments. Following a request from the European Medicines Agency, consensus was reached on the temporary prioritization of patients for treatment based on disease severity and potential reversibility. Advice on follow-up of patients was agreed upon. This consensus is proposed to support the temporary guidelines issued throughout the period of ERT shortage, which will most likely last until April 2011",

author = "Linthorst, \{Gabor E.\} and Germain, \{Dominique P.\} and Hollak, \{Carla E. M.\} and Derralynn Hughes and Arndt Rolfs and Christoph Wanner and Atul Mehta",

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AU - Linthorst, Gabor E.

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AU - Hollak, Carla E. M.

AU - Hughes, Derralynn

AU - Rolfs, Arndt

AU - Wanner, Christoph

AU - Mehta, Atul

PY - 2011

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AB - The shortage of enzyme for treatment of Fabry disease has caused anxiety among patients, physicians and governments. Following a request from the European Medicines Agency, consensus was reached on the temporary prioritization of patients for treatment based on disease severity and potential reversibility. Advice on follow-up of patients was agreed upon. This consensus is proposed to support the temporary guidelines issued throughout the period of ERT shortage, which will most likely last until April 2011

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SP - 99

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JO - Molecular genetics and metabolism

JF - Molecular genetics and metabolism

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Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT)

Abstract

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