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

ther driving force. They are directly funding some research, and they're working smarter and getting creative about drawing attention and dollars for research. Deeper insights into the genetics of these disorders and federal legislation extending patent protection for these medicines also have helped.

Jarvis spent time with several families of children dealing with rare diseases, saw their passion first-hand and includes their stories in the article.

It describes how venture capital firms have become engaged in funding research on rare diseases to the point where more venture capital money may be going toward drugs for rare diseases than for any other type of medication, except for cancer drugs.

Most of the 7,000 rare diseases result from genetic defects, and about half of those affect children. In some cases, children die before they reach adulthood without treatment. Many of the rarest of these diseases affect only a few dozen to a few thousand people. Hunter syndrome, for instance, affects fewer than 500 boys in the U.S., and Gaucher's disease affects about 5,000 people.

Misdiagnosis is a common theme, Jarvis writes. It takes the typical patient 7.6 years and consultations with eight physicians to finally discover what's really going on.

Jarvis reports that until recently, pharmaceutical companies often focused on "blockbuster" drugs, medicines for common conditions like high cholesterol, high blood pressure and diabetes that would have annual sales of at least $1 billion. However, companies like Genzyme, a subsidiary of Sanofi, showed that medications for rare diseases can make money by pricing them high enough to cover research and development costs, and having insurance companies reimburse most patient costs. That's an attractive scenario for larger companies, which are getting into the market, even creating special units focused on this segment, Jarvis adds.

Scientists know more about rare diseases tha
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Contact: Michael Bernstein
m_bernstein@acs.org
202-872-6042
American Chemical Society
Source:Eurekalert

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