Abstract：OBJECTIVE To summarize the latest research progress in the development of enzyme replacement therapy drugs for rare diseases at home and abroad and their pharmaceutical assessment. METHODS Based on the representative literature at home and abroad, this paper propose key points for consideration (such as molecular design, host selection, production process and quality control, etc.), as well as comments and suggestions for researcher of such products from review and evaluation perspective. RESULTS Due to the complexity and heterogeneity of the drug molecules of enzyme replacement therapy for rare diseases, the quality research is the main difficulty in pharmaceutical development and evaluation. CONCLUSION In recent years, in order to alleviate the lack of rare disease drugs in China and meet the urgent clinical needs, Chinese government has issued a series of policies to encourage the research and development of rare disease drugs.
程速远, 寇雅真, 罗建辉. 罕见病酶替代疗法药物研究进展及药学评价思考[J]. 中国药学杂志, 2020, 55(13): 1128-1132.
CHENG Su-yuan, KOU Ya-zhen, LUO Jian-hui. Research Progress of Enzyme Replacement Therapy for Rare Diseases Products and Their Pharmaceutical Assessment. Chinese Pharmaceutical Journal, 2020, 55(13): 1128-1132.
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