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Orphan Drugs and Rare Diseases (Electronic book text)
Loot Price: R7,143
Discovery Miles 71 430
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Orphan Drugs and Rare Diseases (Electronic book text)
Series: Drug Discovery, Volume 38
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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; however, individually, any single, rare disease may
only affect a handful of people making them commercially
unattractive for the biopharmaceutical industry to target. Ground
breaking legislation, starting with the Orphan Drug Act that was
passed in the US in 1983 to provide financial incentives for
companies to develop orphan drugs, has sparked ever increasing
interest from biopharmaceutical companies to tackle rare diseases.
These developments have made rare diseases, and the orphan drugs
that treat them, sufficiently attractive to pharmaceutical
development and many pharmaceutical companies now have research
units dedicated to this area of research. It is therefore timely to
review the area of orphan drugs and some of the basic science, drug
discovery and regulatory factors that underpin this important, and
growing, area of biomedical research. Written by a combination of
academic and industry experts working in the field, this text
brings together expert authors in the regulatory, drug development,
genetics, biochemistry, patient advocacy group, medicinal chemistry
and commercial domains to create a unique and timely reference for
all biomedical researchers interested in finding out more about
orphan drugs and the rare diseases they treat. Providing an
up-to-date monograph, this book covers the basic science, drug
discovery and regulatory elements behind orphan drugs and will
appeal to medicinal and pharmaceutical chemists, biochemists and
anyone working within the fields of rare disease research and drug
development or pharmaceuticals in industry or academia.
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