Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT)
Copyright © 2010 Elsevier Inc. All rights reserved..
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.
| Errataetall: |
CommentIn: Mol Genet Metab. 2011 Jan;102(1):4-5. - PMID 21111645 |
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| Medienart: |
E-Artikel |
| Erscheinungsjahr: |
2011 |
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| Erschienen: |
2011 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:102 |
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| Enthalten in: |
Molecular genetics and metabolism - 102(2011), 1 vom: 01. Jan., Seite 99-102 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Linthorst, Gabor E [VerfasserIn] |
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| Links: |
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| Themen: |
Agalsidase beta |
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| Anmerkungen: |
Date Completed 25.04.2011 Date Revised 31.03.2022 published: Print-Electronic CommentIn: Mol Genet Metab. 2011 Jan;102(1):4-5. - PMID 21111645 Citation Status MEDLINE |
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| doi: |
10.1016/j.ymgme.2010.11.155 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| 520 | |a 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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