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摘要: 罕见病是一类具有发病率极低、患者总数少等特点的疾病，治疗罕见病的药物称为孤儿药。目前已知的皮肤罕见病约450种，大多数疾病缺乏有效的治疗手段，在国家的政策支持和社会各界的积极推动下，近年来皮肤罕见病的治疗药物不断涌现，本文将梳理目前国内外已获批上市的皮肤孤儿药及中国用药可及性现状，以提高对皮肤罕见病及孤儿药的认识。Abstract: Rare diseases are characterized by extremely low incidence rate and small number of patients in total. The drugs used to treat rare diseases are called orphan drugs. Currently, 450 kinds of rare skin diseases have been identified, most of which lack in effective treatments. Supported by the policy-making from the country and pushed by all sectors of the society, drugs for rare skin diseases have been emerging continuously recently in China. This paper reviews the current accessibility of the orphan drugs for skin rare diseases that have been approved in China and in other countries and regions for a better understanding of rare dermatosis and orphan drugs for the diseases.
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Key words:
- rare skin diseases /
- orphan drugs /
- rare disease drugs /
- accessibility
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表 1 生物制剂治疗化脓性汗腺炎相关临床研究
Table 1. Clinical researches on the treatment of hidradenitis suppurativa with biologics
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表 2 生物制剂治疗红皮病型银屑病相关临床研究
Table 2. Clinical researches on the treatment of erythrodermic psoriasis with biologics
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