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

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.