Renaissance in New Drugs for Rare Diseases Reported

Once famously described as "orphan diseases, too small to be noticed, too small to be funded" in the Hollywood film “Lorenzo's Oil,” rare diseases are getting unprecedented attention today among drug manufacturers, who are ramping up research efforts and marketing new medicines that promise fuller lives for children and other patients with these heartbreaking conditions. That's the conclusion of a major examination, published as the cover story of the May 13, 2013 issue of Chemical & Engineering News (C&EN), the weekly newsmagazine of the world's largest scientific society (the American Chemical Society--ACS), of the status of new drugs for the 7,000 conditions that affect 200,000 or fewer patients and fall into the "rare disease" category. The article was written by senior editor Lisa Jarvis after months of interviews with patients, parents, pharmaceutical industry officials, and others. C&EN reaches more than 138,000 scientists, policy-makers, educators, and others around the world. "For most of the last century, people afflicted by rare diseases — especially the parents and families of young children — shared the heartbreak of knowing that medicines to treat their loved ones were little more than a dream," says A. Maureen Rouhi, Ph.D., editor-in-chief of C&EN. "As our story documents in such compelling fashion, that situation is dramatically changing. Pharmaceutical companies are making unprecedented investments in medicines for these enigmatic conditions, popularized in films, and treatments for some are on the way." Jarvis describes how a combination of factors has coalesced to foster a renaissance in drug development for rare diseases.
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