Orphan Drugs and Rare Diseases
Gebundenes Buch

Orphan Drugs and Rare Diseases

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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.