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, but individually, any single rare disease may only affect a handful of people making them unattractive for the biopharmaceutical industry to target. Providing an up-to-date mongraph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs.
Inhaltsverzeichnis
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
