寻求批准：寻常型天疱疮的现有及未来疗法
Seeking approval: present and future therapies for pemphigus vulgaris.
作者信息
Mao Xuming, Payne Aimee S
机构信息
University of Pennsylvania, Department of Dermatology, 217A Clinical Research Building, 415 Curie Boulevard, Philadelphia, PA 19104, USA.
出版信息
Curr Opin Investig Drugs. 2008 May;9(5):497-504.
Abstract
Pemphigus vulgaris is an autoimmune blistering disease of the skin and mucous membranes. Despite the potentially fatal prognosis, there are currently no FDA-approved treatments specifically for pemphigus. In 2006, the FDA designated orphan drug status to mycophenolate mofetil for the treatment of pemphigus vulgaris, indicating both federal and commercial interest in developing therapies for this devastating disease. This review focuses on pemphigus therapies that are currently in preclinical or clinical trials, as well as potential novel therapies based on recent advances in the understanding of the pathophysiology of this disease.
摘要
寻常型天疱疮是一种皮肤和黏膜的自身免疫性水疱病。尽管预后可能致命，但目前尚无美国食品药品监督管理局（FDA）批准的专门用于治疗天疱疮的疗法。2006年，FDA指定霉酚酸酯用于治疗寻常型天疱疮的孤儿药地位，这表明联邦政府和商业机构都有兴趣开发针对这种毁灭性疾病的治疗方法。本综述重点关注目前处于临床前或临床试验阶段的天疱疮治疗方法，以及基于对该疾病病理生理学理解的最新进展的潜在新型疗法。
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