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 |
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Pages (from-to) | 99-102 |
Journal | Molecular Genetics and Metabolism |
Volume | 102 |
Issue number | 1 |
DOIs | |
Publication status | Published - 2011 |