Department of Internal Medicine, Division of Endocrinology and Metabolism, Academic Medical Center, Amsterdam, The Netherlands.
Mol Genet Metab. 2011 Jan;102(1):99-102. doi: 10.1016/j.ymgme.2010.11.155. Epub 2010 Nov 30.
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.
治疗法布里病的酶短缺问题引起了患者、医生和政府的焦虑。在接到欧洲药品管理局的请求后,根据疾病严重程度和潜在可逆性,各方就临时确定治疗优先顺序达成了共识。还就患者的后续处理达成了一致意见。提出该共识是为了支持 ERT 短缺期间发布的临时指南,该指南很可能会持续到 2011 年 4 月。