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

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 today in the weekly newsmagazine of the world's largest scientific society, of the status of new drugs for the 7,000 conditions that affect 200,000 patients or fewer and fall into the "rare disease" category. Written by senior editor Lisa Jarvis after months of interviews with patients, parents, pharmaceutical industry officials and others, it is the cover story in this week's issue of Chemical & Engineering News (C&EN), which reaches more than 138,000 scientists, policy-makers, educators and others. The 4-part story, and a wealth of associated online-only content, is available at the end of this press release.

"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.

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, Jarvis explains in the article. Patient advocacy groups are ano
'/>"/>

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

Page: 1 2 3 4 5

Related biology news :

1. Newly released FDA draft guidelines for biosimilar drugs to be discussed at conference
2. Nanopills release drugs directly from the inside of cells
3. Genetic variation in East Asians found to explain resistance to cancer drugs
4. Discovery provides blueprint for new drugs that can inhibit hepatitis C virus
5. Internet and new drugs: A challenge for public health
6. Study raises questions about use of anti-epilepsy drugs in newborns
7. Drugs from lizard saliva reduces the cravings for food
8. A new method detects traces of veterinary drugs in baby food
9. La Jolla Institute discovery could lead to new way to screen drugs for adverse reactions
10. MIT-designed cooler preserves tuberculosis drugs, records doses
11. Arsenic for better drugs and cleaner crops
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/15/2014)... spreading rapidly and to an unexpected extent. The outbreak ... and the virus shows a new disease dynamic in ... this reason, the German National Academy of Sciences Leopoldina, ... and the Union of the German Academies of Sciences ... epidemic today. , In the statement the academies call ...
(Date:10/15/2014)... NXT-ID, Inc. (NASDAQ: NXTD and NXTDW) ... growing mobile commerce market releases photos and video of the recent ... October 13 th . Gino Pereira , ... angel investor Mr. Chad A. Verdi rang the bell.  ... his investors and employees "for their work and dedication in bringing ...
(Date:10/15/2014)... 15, 2014   Neurotechnology , a provider ... availability of the VeriLook Surveillance 3.0 software ... real-time biometric face identification using live video streams ... The new version not only identifies faces in ... objects while they are moving through the video ...
Breaking Biology News(10 mins):Academies call for consequences from the Ebola virus epidemic 2Academies call for consequences from the Ebola virus epidemic 3Academies call for consequences from the Ebola virus epidemic 4Academies call for consequences from the Ebola virus epidemic 5Photo Release: NXT-ID Inc. Rings Opening Bell of the NASDAQ Stock Market October 13th 2Photo Release: NXT-ID Inc. Rings Opening Bell of the NASDAQ Stock Market October 13th 3VeriLook Surveillance 3.0 SDK Identifies Faces and Moving Objects, Differentiates Pedestrians from Other Moving Objects in Video Surveillance Systems 2VeriLook Surveillance 3.0 SDK Identifies Faces and Moving Objects, Differentiates Pedestrians from Other Moving Objects in Video Surveillance Systems 3
... Dartmouth researchers use individual genetic data to reveal the powers ... influences human health and what genes can reveal about the ... in the Sept. 11 issue of the American Journal ... Professor Scott Williams analyzed how personal genetic testing companies are ...
... turning waste biomass such as dead trees, agricultural ... to power conventional engines has been licensed by the ... LLC, a private biofuels company based in Sacramento, Calif. ... industry: the ultra-low-sulfur biofuel does not require additional refinement ...
... is one of the most common neuropsychiatric disorders ... with the disorder. Key symptoms of ADHD include ... ability to focus attention. Clinically, three different ADHD ... primarily hyperactive/impulsive subtype, and a combined subtype in ...
Cached Biology News:Dartmouth researchers get personal with genetics 2Dartmouth researchers get personal with genetics 3UGA licenses technology to make fuel from dead forests and agricultural waste 2The role of genetic factors in adult ADHD 2The role of genetic factors in adult ADHD 3The role of genetic factors in adult ADHD 4The role of genetic factors in adult ADHD 5The role of genetic factors in adult ADHD 6
(Date:10/22/2014)... Md. (PRWEB) October 21, 2014 Shimadzu ... an LC/MS/MS data tool for the pain management and ... of data processing and review by allowing users to ... to clinicians. , QuantAnalytics allows analysts to filter ... This expedites data processing by eliminating the need to ...
(Date:10/22/2014)... Hill, SC (PRWEB) October 22, 2014 ... expansion of its cardiovascular pharmacogenetics menu, which enables ... patient outcomes. With PCLS’s evidence-based results, healthcare ... needs and optimize their therapy, while minimizing risks ... In the U.S., according to the FDA [1] ...
(Date:10/22/2014)... Research and Markets  has announced the addition of ... Geography - Global Analysis and Forecast (2014 - 2020)" ... electronics, also called as polymer electronics or plastic electronics, ... small conductive molecules and electrically conductive polymers. It is ... carbon based, made using synthetic strategies developed in the ...
(Date:10/20/2014)... 2014 Asterias Biotherapeutics, Inc. (NYSE MKT: ... a Notice of Grant Award (NGA) with the ... 1, 2014.  The NGA provides for the immediate ... of additional grant funds pursuant to the previously ... development of Asterias, product, AST-OPC1. The grant provides ...
Breaking Biology Technology:Shimadzu Releases QuantAnalytics Open Access LCMS Software Package for Clinical Applications 2PCLS Announces Expansion of Cardiovascular Pharmacogenetic Testing Menu 2PCLS Announces Expansion of Cardiovascular Pharmacogenetic Testing Menu 3Global Organic Electronics (Semiconductor, Conductive, Dielectric and Substrate) Market - Analysis and Forecast (2014 - 2020) 2Global Organic Electronics (Semiconductor, Conductive, Dielectric and Substrate) Market - Analysis and Forecast (2014 - 2020) 3Asterias Biotherapeutics Announces Notice of Grant Award with CIRM for Phase 1/2a Clinical Trial of AST-OPC1 in Complete Cervical Spinal Cord Injury 2Asterias Biotherapeutics Announces Notice of Grant Award with CIRM for Phase 1/2a Clinical Trial of AST-OPC1 in Complete Cervical Spinal Cord Injury 3Asterias Biotherapeutics Announces Notice of Grant Award with CIRM for Phase 1/2a Clinical Trial of AST-OPC1 in Complete Cervical Spinal Cord Injury 4
... 7 /PRNewswire-FirstCall/ - Oncothyreon Inc. (the,"Company") (Nasdaq: ... development of innovative therapeutic products for the treatment ... its fourth quarter and,full year 2007 financial results ... the U.S. and Canadian markets. Following the release, ...
... Corporation,(Nasdaq: ACEL ) today reported its financial results ... recorded a net loss of approximately $1.7 million, or ... fiscal quarter ended,Jan. 31, compared to a net loss ... share, for the second quarter of fiscal 2007. The ...
... PARK, Ill., March 7 Abbott (NYSE: ... Annual Global Healthcare,Conference on Tuesday, March 18, 2008. ... financial officer, will make a formal,presentation on the ... live audio webcast of the presentation will be ...
Cached Biology Technology:Alfacell Reports Financial Results for Second Quarter of Fiscal 2008 2Alfacell Reports Financial Results for Second Quarter of Fiscal 2008 3Alfacell Reports Financial Results for Second Quarter of Fiscal 2008 4Alfacell Reports Financial Results for Second Quarter of Fiscal 2008 5
Liquid. PBS containing 10 mM NaN 3 . AVOID FREEZE/THAW CYCLES. Recognizes α1, 2, 3, 5, 6 and 7 subunits of 20S proteasome. ...
Mouse monoclonal antibody to Lats2....
... Membrane Protein 2, Synaptobrevin 2). ... homology with any other known ... expected that the antibody will ... multata and bovine due to ...
Human GCP-2 is a member of the CXC family of chemokines. It also promotes neutrophil chemotaxis and deganulation. GF063 is an 8.0 kDa protein containing 73 amino acid residues....
Biology Products: