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Renaissance in new drugs for rare diseases: Report in world's largest scientific socity magazine
Date:5/13/2013

ss and funds.

Case Hogan is a rambunctious six-year-old with Hunter syndrome, who is participating in a clinical trial of Shire's HGT2310, an enzyme replacement therapy delivered into his spinal cavity. After two and a half years in the study, his mother, Melissa Hogan, says his behavior and mental abilities have improved dramatically.

Jill Wood's son, Jonah, has Sanfilippo syndrome, another type of MPS disease that affects only a few dozen children in the U.S. She speaks with her local representatives, congressional staffers, the media and scientists about research and funding. Wood has started a nonprofit organization called "Jonah's Just Begun," as well as a company called Phoenix Nest to develop promising drug candidates for Sanfilippo syndrome if companies don't step up to support them.

Among highlights from the story:

  • Misdiagnosis is common. It can take an average of 7.6 years and eight physicians for people with a rare disease to discover what's really going on.

  • Big drug companies, long focused on producing blockbuster drugs for common conditions like high blood pressure and high cholesterol, are adopting new business models that will make rare-disease drug production feasible.

  • Medications for rare diseases can exceed $200,000 per year. Insurance companies typically cover most of the costs. The potential for big profits on expensive drugs is an incentive for companies to get into the game.

  • Several big-name blockbuster drugs are going "off-patent," leading to increased competition from generic versions and lower profits. Fewer new blockbuster drugs are in the works. These factors also are making the rare-disease market attractive.

  • Companies are realizing that some drugs for orphan diseases can help patients with more common ones, which makes such medicines even more attractive for further development. For example, patients with Sanfilippo syndrome and those
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Contact: Michael Bernstein
m_bernstein@acs.org
202-872-6042
American Chemical Society
Source:Eurekalert

Page: 1 2 3 4 5

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