Orphan Drugs and Rare Diseases (eBook)

UK) Palmer Michael J (Medicinal Chemistry Consultant, UK) Pryde David (Pfizer (Herausgeber)

eBook Download: PDF

2014
350 Seiten
Royal Society of Chemistry (Verlag)
978-1-78262-420-2 (ISBN)

Infos

This book provides an up-to-date monograph on the drug discovery and regulatory elements of therapuetics used to treat rare or "orphan" diseases.

Inhalt

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®) for the Treatment of Mucopolysaccharidosis II (Hunter Syndrome); Discovery and Development of ILARIS® 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®; 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

Produkt-Details

• Erscheint lt. Verlag: 30.7.2014
• Reihe/Serie: ISSN
• Reihe/Serie: ISSN
• Verlagsort: Cambridge
• Sprache: englisch
• Maße: 160 x 160 mm
• Themenwelt: Medizin / Pharmazie ► Gesundheitsfachberufe
• Themenwelt: Medizin / Pharmazie ► Medizinische Fachgebiete ► Pharmakologie / Pharmakotherapie
• Themenwelt: Naturwissenschaften ► Biologie ► Biochemie
• Schlagworte: Discovery • Diseases • Drugs • orphan • Rare
• ISBN-10: 1-78262-420-1 / 1782624201
• ISBN-13: 978-1-78262-420-2 / 9781782624202