THE CHEMISTRY OF DRUGS

By BARBARA ANDERSON

CONTENTS CHAPTER 1 DEFINITIONS, HISTORY AND REGULATORY FRAMEWORK FOR RARE DISEASES AND ORPHAN DRUGS CHAPTER 2 DIAGNOSIS OF RARE INHERITED DISEASES CHAPTER 3 THE CHALLENGES OF CONDUCTING CLINICAL TRIALS IN DISEASES WITH SMALL TARGET POPULATIONS CHAPTER 4 TREATING RARE DISEASES: BUSINESS MODEL FOR ORPHAN DRUG DEVELOPMENT CHAPTER 5 DISEASE ADVOCACY ORGANISATIONS CHAPTER 6 PHARMACOLOGICAL CHAPERONES TO CORRECT ENZYME FOLDING, CELLULAR TRAFFICKING AND LYSOSOMAL ACTIVITY CHAPTER 7 DISCOVERY AND CLINICAL DEVELOPMENT OF IDURSULFASE (ELAPRASE®) FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSIS II (HUNTER SYNDROME) CHAPTER 8 DISCOVERY AND DEVELOPMENT OF ILARIS® FOR THE TREATMENT OF CRYOPYRIN- ASSOCIATED PERIODIC SYNDROMES CHAPTER 9 DISCOVERY AND DEVELOPMENT OF TAFAMIDIS FOR THE TREATMENT OF TTR FAMILIAL AMYLOID POLYNEUROPATHY CHAPTER 10 SMALL MOLECULES THAT RESCUE F508DEL CFTR AS CYSTIC FIBROSIS THERAPIES CHAPTER 11 DRUG DISCOVERY APPROACHES FOR RARE NEUROMUSCULAR DISEASES CHAPTER 12 UNLEASHING THE POWER OF SEMI-SYNTHESIS: THE DISCOVERY OF TORISEL® CHAPTER 13 TREATMENTS FOR PULMONARY ARTERIAL HYPERTENSION CHAPTER 14 SOLIRIS (ECULIZUMAB): DISCOVERY AND DEVELOPMENT CHAPTER 15 THE DISCOVERY AND DEVELOPMENT OF RUXOLITINIB FOR THE TREATMENT OF MYELOFIBROSIS CHAPTER 16 POSSIBLE SOLUTIONS TO ACCELERATE ACCESS TO RARE DISEASE TREATMENTS