Orphan Drugs: From Regulatory Issues to Clinical Transition (Hardcover)

Introduction. Policies and Regulatory affairs. Public industry partnership. Advances in NGS and its impact on orphan drug discoveries. Example from rare disorders: SMA- from basic mechanism to phase 3 trial. Duchene Muscular Dystrophy: clinical and therapeutic overview (focusing on current drug pipeline). Other Muscular Dystrophies: Clinical overview. Hereditary Neuropathies. Amyotrophic lateral sclerosis: Current therapeutic pipeline. Familial Dysautonomia. Pamidronate for Rare bone diseases. Repurposing orphan drugs. Orphan Diseases in middle east: Clinical overview. Clinical trials for rare diseases- Lessons learned along with future directions.