Orphan drugs and rare diseases
著者
書誌事項
Orphan drugs and rare diseases
(RSC drug discovery series / editor-in-chief, David Thurston ; series editors, David Fox ... [et al.], no. 38)
Royal Society of Chemistry, c2014
大学図書館所蔵 全1件
  青森
  岩手
  宮城
  秋田
  山形
  福島
  茨城
  栃木
  群馬
  埼玉
  千葉
  東京
  神奈川
  新潟
  富山
  石川
  福井
  山梨
  長野
  岐阜
  静岡
  愛知
  三重
  滋賀
  京都
  大阪
  兵庫
  奈良
  和歌山
  鳥取
  島根
  岡山
  広島
  山口
  徳島
  香川
  愛媛
  高知
  福岡
  佐賀
  長崎
  熊本
  大分
  宮崎
  鹿児島
  沖縄
  韓国
  中国
  タイ
  イギリス
  ドイツ
  スイス
  フランス
  ベルギー
  オランダ
  スウェーデン
  ノルウェー
  アメリカ
注記
Includes bibliographical references and index
Also issued online
内容説明・目次
内容説明
Orphan drugs are designated drug substances that are intended to treat rare or 'orphan' diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world's population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target.
Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan drugs, has sparked ever increasing interest from biopharmaceutical companies to tackle rare diseases. These developments have made rare diseases, and the orphan drugs that treat them, sufficiently attractive to pharmaceutical development and many pharmaceutical companies now have research units dedicated to this area of research. It is therefore timely to review the area of orphan drugs and some of the basic science, drug discovery and regulatory factors that underpin this important, and growing, area of biomedical research.
Written by a combination of academic and industry experts working in the field, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat.
Providing an up-to-date monograph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs and will appeal to medicinal and pharmaceutical chemists, biochemists and anyone working within the fields of rare disease research and drug development or pharmaceuticals in industry or academia.
目次
- Introduction
- Foreword on Rare Diseases
- Definitions, History and Regulatory Framework for Rare Diseases and Orphan Drugs
- Diagnosis of Rare Inherited Diseases
- Treating Rare Diseases: Business Model For Orphan Drug Development
- Disease Advocacy Organizations
- Lysosomal Storage Diseases
- Discovery and Clinical Development of Idursulfase (Elaprase (R)) for the Treatment of Mucopolysaccharidosis II (Hunter Syndrome)
- Discovery and Development of ILARIS (R) for the Treatment of Cryopyrin-Associated Periodic Syndromes
- Discovery and Development of Tafamidis for the Treatment of TTR Familial Amyloid Polyneuropathy
- Small Molecules that Rescue F508del CFTR as Cystic Fibrosis Therapies
- Rare Neuromuscular Diseases
- Unleashing the Power of Semi-synthesis: The Discovery of Torisel (R)
- Treatments for Pulmonary Arterial Hypertension
- Soliris (Eculizumab): Discovery and Development
- The Discovery and Development of Ruxolitinib for the Treatment of Myelofibrosis
- Possible Solutions to Accelerate Access to Rare Disease Treatments
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