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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 worlds 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.
David C. Pryde is a medicinal chemist based at Pfizer Global Research and Development Neusentis research unit. He earned his B.Sc. in Pure and Applied Chemistry from the University of Strathclyde, Scotland (1991), PhD from the University of Nottingham, England, under the supervision of Professor Gerry Pattenden in the field of biomimetic radical cyclisations (1994) and carried out postdoctoral research with Professor Albert Meyers at Colorado State University on stereoselective metallation chemistry before joining Pfizer in 1997. He has worked in several therapeutic areas from cardiovascular and pain, to sexual health and anti-infectives and identified development candidates in each of these areas. His research interests include all aspects of medicinal chemistry, synthetic biologics and chemical biology methodology. He has coauthored more than 50 papers and patents.
Michael J Palmer is a medicinal chemist currently specialising in Neglected Disease Research, and in particular malaria. He earned his B.Sc. in chemistry at the University of Nottingham in 1975 and his M.Sc. from the University of East Anglia in 1981 under the supervision of Pr. Alan Katritzky and Pr. Sandy McKillop. He worked for Pfizer Global R and D, Sandwich for 36 years, spanning many therapeutic areas and during which time he was a leading part of chemistry teams that helped to take the antihypertensive alpha blocker CarduraTM to market and 6 drugs to the clinic, including the once daily PDE5 agent PF-489791, cl“/
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