Navigation Links
Renaissance in new drugs for rare diseases
Date:5/15/2013

Once famously described as "orphan diseases, too small to be noticed, too small to be funded" in the Hollywood drama "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 finding of a major examination, published in the current edition of Chemical & Engineering News. C&EN is the weekly newsmagazine of the American Chemical Society, the world's largest scientific society, and reaches more than 138,000 scientists, policymakers, educators and others. Written by Senior Editor Lisa Jarvis after months of interviews with patients, parents, pharmaceutical industry officials and others, the story focuses on the status of new drugs for rare diseases. Those 7,000 conditions affect 200,000 patients or fewer.

Jarvis describes how a combination of factors coalesced to foster a renaissance in drug development for rare diseases. Smaller drug companies, for instance, have shown that it is possible to make big profits from sales of medicines for rare diseases, and larger companies have taken notice. Patient advocacy groups are another driving force. They are directly funding some research and drawing attention and dollars for research. Deeper insights into the genetics of rare diseases and federal legislation extending patent protection for treatments also have helped.


'/>"/>

Contact: Michael Bernstein
m_bernstein@acs.org
202-872-6042
American Chemical Society
Source:Eurekalert

Page: 1

Related biology news :

1. Renaissance in new drugs for rare diseases: Report in worlds largest scientific socity magazine
2. Newly released FDA draft guidelines for biosimilar drugs to be discussed at conference
3. Nanopills release drugs directly from the inside of cells
4. Genetic variation in East Asians found to explain resistance to cancer drugs
5. Discovery provides blueprint for new drugs that can inhibit hepatitis C virus
6. Internet and new drugs: A challenge for public health
7. Study raises questions about use of anti-epilepsy drugs in newborns
8. Drugs from lizard saliva reduces the cravings for food
9. A new method detects traces of veterinary drugs in baby food
10. La Jolla Institute discovery could lead to new way to screen drugs for adverse reactions
11. MIT-designed cooler preserves tuberculosis drugs, records doses
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/31/2016)... RATON, Florida , March 31, 2016 ... LEGX ) ("LegacyXChange" or the "Company") ... for potential users of its soon to be launched ... video ( https://www.youtube.com/channel/UCyTLBzmZogV1y2D6bDkBX5g ) will also provide ... the use of DNA technology to an industry that ...
(Date:3/29/2016)... BOCA RATON, Florida , March 29, 2016 /PRNewswire/ ... ("LegacyXChange" or the "Company") LegacyXChange "LEGX" and SelectaDNA/CSI Protect ... Synthetic DNA in ink used in a variety of ... preventing theft. Buyers of originally created collectibles from athletes ... authenticity through forensic analysis of the DNA. ...
(Date:3/22/2016)... , March 22, 2016 ... research report "Electronic Sensors Market for Consumer Industry by ... & Others), Application (Communication & IT, Entertainment, ... - Global Forecast to 2022", published by ... is expected to reach USD 26.76 Billion ...
Breaking Biology News(10 mins):
(Date:6/27/2016)... ... 2016 , ... Newly created 4Sight Medical Solutions ... healthcare market. The company's primary focus is on new product introductions, to include ... are necessary to help companies efficiently bring their products to market. , The ...
(Date:6/24/2016)... , June 24, 2016  Regular discussions on a ... take place between the two entities said Poloz. ... Ottawa , he pointed to the country,s ... the federal government. ... said, "Both institutions have common economic goals, why not sit ...
(Date:6/23/2016)... ... June 23, 2016 , ... Mosio, a leader in clinical ... Patient Recruitment and Retention Tips.” Partnering with experienced clinical research professionals, Mosio revisits ... tips, tools, and strategies for clinical researchers. , “The landscape of how patients ...
(Date:6/23/2016)... , June 23, 2016 A person commits ... the crime scene to track the criminal down. ... U.S. Food and Drug Administration (FDA) uses DNA evidence to ... Sound far-fetched? It,s not. The FDA has ... to support investigations of foodborne illnesses. Put as simply as ...
Breaking Biology Technology: