期刊
MOLECULAR GENETICS AND METABOLISM
卷 102, 期 1, 页码 99-102出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.ymgme.2010.11.155
关键词
Fabry disease; Enzyme replacement therapy; EMA; Consensus
资金
- Genzyme Corporation
- Shire HGT
- Shire
- Actelion
- Amicus Therapeutics
- Genzyme
- Amicus
- Shire Genzyme
- Biomarin
- Fabry Registry European Board of advisors
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. (C) 2010 Elsevier Inc. All rights reserved.
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