罕见病的蛋白质替代疗法：监管审批的轻松之路？
Protein replacement therapies for rare diseases: a breeze for regulatory approval?
机构信息
Laurelwood Biopartners, Boston, MA 02115, USA.
出版信息
Sci Transl Med. 2013 Mar 27;5(178):178fs10. doi: 10.1126/scitranslmed.3005007.
PMID:23536010
Abstract
Protein replacement therapies for rare monogenic diseases have a higher probability of regulatory approval compared with biologics, small molecules, and grant-funded orphan drugs.
摘要
与生物制剂、小分子药物和资助性孤儿药相比，用于罕见单基因疾病的蛋白质替代疗法获得监管批准的可能性更高。
Zotero 插件