Small molecule therapy for genetic disease / edited by Jess G. Thoene.

• Material Type: 図書
• ISBN: 9780521517812 (hardback); 0521517818 (hardback)
• Title: Small molecule therapy for genetic disease / edited by Jess G. Thoene.
• Author Heading: Thoene, Jess G.
• Publication, Distribution, etc.: Cambridge, UK : Cambridge University Press; New York : Cambridge University Press
• Publication Date: 2010.
• Publication Date (W3CDTF): 2010
• Extent: xiii, 223 p. : ill. (some col.) ; 27 cm.
• Place of Publication (Country Code): GB
• Text Language Code: eng
• Subject Heading: Metabolism, Inborn errors of -- Chemotherapy -- Handbooks, manuals, etc.; Metabolism, Inborn errors of -- Gene therapy -- Handbooks, manuals, etc.; Genetic disorders -- Chemotherapy -- Handbooks, manuals, etc.; Genetic Diseases, Inborn -- drug therapy.; Orphan Drug Production.; Rare Diseases -- drug therapy.; Small Molecule Libraries -- therapeutic use.
• LCC: RC627.8
• DDC: 616.3/9042
• NDLC: SC2; SC311
• Note (Bibliography): Includes bibliographical references and index.
• Holding library: 国立国会図書館
• Call No.: SC2-B89
• Data Provider (Database): 国立国会図書館 : 国立国会図書館蔵書; https://ndlsearch.ndl.go.jp
• Bibliographic ID (NDL): 000011053877; http://id.ndl.go.jp/bib/000011053877
• LC Control Number: 2010002899; https://lccn.loc.gov/2010002899
• OCLC No.: 502393232; https://www.worldcat.org/oclc/502393232
• Cataloging Rule: Anglo-American Cataloging Rules
• Bibliographic Record Category (NDL): 211

• Contents: Machine generated contents note: 1. FDA and the Regulation of Small Molecules for Orphan Diseases Marlene Haffner and Tan Nguyen; 2. The office of rar...
• Summary, etc.: "This book summarizes the substantial work that has been accomplished in the treatment of inborn errors of metabolism with simple molecules. These age...