Adult Metabolic Diseases Clinic, Level 4—2775 Laurel Street, Vancouver, BC, V5Z 1M9, Canada.
Mol Genet Metab. 2011 Jan;102(1):4-5. doi: 10.1016/j.ymgme.2010.11.002. Epub 2010 Dec 15.
The recent shortages of enzyme replacement therapy for Fabry disease have highlighted areas of vulnerability for patients who require this treatment. Guidelines on allocation of limited stock of enzyme replacement therapy are of use for clinicians dealing with the current shortages. However, the community of metabolic physicians must advocate for changes that will minimize the impact of future drug shortages for their patients with lysosomal storage diseases.
最近法布瑞病（Fabry disease）的酶替代疗法出现短缺，凸显了需要接受这种治疗的患者存在脆弱性的领域。关于有限库存的酶替代疗法分配的指南，对于处理当前短缺的临床医生是有用的。然而，代谢科医生群体必须倡导做出改变，以尽量减少未来药物短缺对他们的溶酶体贮积病患者的影响。
