Lee David K, Wong Barbara
J Popul Ther Clin Pharmacol. 2014;21(1):e42-6. Epub 2014 Feb 23.
An orphan drug is a pharmaceutical or biological drug that is used to treat a rare disease. The development of a Canadian orphan drug framework is intended to help translate scientific discoveries into meaningful results for Canadian rare disease patients.
孤儿药是一种用于治疗罕见病的药物或生物制剂。加拿大孤儿药框架的制定旨在帮助将科学发现转化为对加拿大罕见病患者有意义的成果。