孤儿药:专题观点

Poluru SriSatya Vani, Vani Mamillapalli, Padmalatha Kantamaneni, Raya Srikeerthi, Thirumalasetty Ruchitharani, Lanka Rajini, Chilakabathini Keziahrani
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引用次数: 0

摘要

根据世界卫生组织的数据,孤儿病是每1000人中有0.65到1人受到影响的疾病。由于这些药物在经济上不稳定且不可行,因此通常不会对其病理生理学或新兴治疗方案进行探索。1983年1月28日,美国制定了一项名为《孤儿药法案》的法律,以促进罕见疾病药物的研究、创造和批准。目前,治疗其他传染病的药物与治疗热带传染病的药物一样多,其中11种药物(4.87%)被认定为孤儿药。一些孤儿药被用于治疗不再符合孤儿药指定要求的疾病。一些企业还制造和生产孤儿药物。核克隆、合理的药物设计和高通量筛选都是药物发现项目中使用的方法,可以导致新药的开发。本文讨论了孤儿药、医学孤儿、罕见疾病、孤儿药法案、孤儿药设计、孤儿药的全球市场情况以及包含孤儿药信息的各种数据库。那些被营利性药物发现工作所忽视的患者对未来充满了希望。
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Orphan Drugs: A Thematic View
Orphan diseases are all medical conditions that impact 0.65 to 1 out of every 1000 people, according to the World Health Organization. Since these are not financially stable and feasible, they are typically not explored for their pathophysiology or the emerging therapy alternatives. On January 28, 1983, in the USA a law known as the Orphan Drug Act was formed to promote the study, creation, and approval of medications for rare diseases. There are currently as many medications for other infectious diseases as there are for tropical infectious diseases, with 11 pharmaceuticals (4.87%) recognized as orphan drugs. Several medications having orphan status are used to treat illnesses that no longer meet the requirements for orphan designation. Some businesses also manufacture and produce orphan medications. Nuclear cloning, rational drug design, and high throughput screening are all methods used in drug discovery programs that could lead to the development of new medicines. This article discusses orphan drugs, medical orphans, rare diseases, the orphan drug act, orphan drug design, the global market scenario of orphan drugs and various databases that contain information about orphan drugs. Patients who have been ignored by for-profit medication discovery efforts have hope for the future.
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