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

n ever before, thanks to progress in genomics and other areas of biomedical science. About 20 years ago, scientists had teased out the molecular basis for fewer than 50 rare diseases. Today, they know the genetic underpinnings for roughly 4,500 it's a complete sea change, which could show scientists the path to develop many more treatments.

On the regulatory side, the Orphan Drug Act of 1983 fostered drug development for rare diseases by granting companies seven years of exclusivity, even when the patent runs out; a waiver for U.S. Food and Drug Administration (FDA) fees; and tax credits. The incentives have worked the number of treatments for these diseases has risen from 10 to over 400 since the Act was created. And just last year, the passage of the FDA Safety Innovation Act made it easier to get them through clinical trials an expensive and challenging hurdle.

Patient advocacy groups, often led by patients themselves or relatives or friends of patients, also are driving the shift. They aren't just attending fundraisers they're taking charge, organizing events and even establishing their own charitable foundations or companies, funding researchers directly. They're telling their stories on social media, lobbying Congress and talking to the media.

To learn more about rare diseases and their impact, Jarvis spent time with patients and their families. She reports about how Jason and Justin Leider, aged 6 and 4, get their "muscle juice," or Elaprase medication, in an IV line in their chests to treat Hunter syndrome. Both children have the rare disease, which affects only boys. Patients lack an important enzyme that breaks down sugars. It is one of the so-called mucopolysaccharidosis (MPS) diseases. Without that enzyme, sugars accumulate in cells across the body, causing a rapid physical and mental decline. Most boys with Hunter syndrome die by age 15.Their parents, Jeff and Deena Leider, founded "Let Them Be Little x2" to raise awarene
'/>"/>

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:4/20/2015)... , April 20, 2015 Huntington Memorial Hospital ... to implant a new miniaturized, wireless monitoring sensor to ... the first and only FDA-approved heart failure monitoring device ... when used by physicians to manage heart failure. ... is implanted in the pulmonary artery (PA) during a ...
(Date:4/17/2015)... April 17, 2015 ... and technological advancement to drive biometric systems market ... 2020   According to a recently ... Systems Market Forecast &  Opportunities, 2020 ", biometric systems market in ... register growth at CAGR of over 22% through ...
(Date:4/14/2015)... Conn. , April 14, 2015 NXT-ID, ... a biometric authentication company focused on the growing mobile ... been nominated for the 2015 New York Design Awards ... The Design100, New York City Design Awards are part ... based on over 2,500 ratings from the marketplace, industry ...
Breaking Biology News(10 mins):Huntington Memorial Hospital Becomes First in the San Gabriel Valley to Offer Patients a New Heart Failure Monitoring Solution 2Huntington Memorial Hospital Becomes First in the San Gabriel Valley to Offer Patients a New Heart Failure Monitoring Solution 3Saudi Arabia Biometric Systems Market to Grow at 22% Through 2020, Says TechSci Research 2Saudi Arabia Biometric Systems Market to Grow at 22% Through 2020, Says TechSci Research 3NXT-ID's Wocket Smart Wallet Nominated for 2015 New York Design Awards in 'Product Design- Technology' 2NXT-ID's Wocket Smart Wallet Nominated for 2015 New York Design Awards in 'Product Design- Technology' 3
... Mexican oregano, all contain apigenin and luteolin, flavonoids that ... inhibiting an important enzyme, according to two new University ... in two aggressive human pancreatic cancer cell lines. But ... cells with apigenin for 24 hours, then applied the ...
... BOSTON (August 15, 2013) -- In a study published ... Molecular Genetics , Drs. Donita Garland, Rosario Fernandez-Godino, and ... Massachusetts Eye and Ear, Harvard Medical School, along with ... genetically engineered to have an inherited form of macular ...
... MEDFORD/SOMERVILLE, Mass. In two recently published papers, ... techniques for predicting the severity of seasonal cholera epidemics ... of accuracy than other methods based on remote ... may provide the essential lead time to strengthen intervention ...
Cached Biology News:Celery, artichokes contain flavonoids that kill human pancreatic cancer cells 2Researchers report a critical role for the complement system in early macular degeneration 2Researchers report a critical role for the complement system in early macular degeneration 3Tufts scientists develop new early warning system for cholera epidemics 2Tufts scientists develop new early warning system for cholera epidemics 3
(Date:5/1/2015)... 2015 Pfenex Inc. (NYSE MKT: PFNX), ... of biosimilar therapeutics including high value and difficult ... quarter 2015 financial results will be released on ... market. At 8:30 am Eastern Time, Pfenex management will ... results and provide a business update. A press release ...
(Date:5/1/2015)... -- According to a new market ... by Type (Monoclonal, Polyclonal), by Technology (Hybridoma), by ... Analysis & Global Forecast to 2019", published by ... Market for the forecast period of 2014 to ... Billion by 2019 from $1.425 Billion in 2014, ...
(Date:4/30/2015)... Cytokinetics, Incorporated (Nasdaq: CYTK) reported total research ... $4.4 million, compared to $8.0 million during the same period ... was $8.9 million, or $0.23 per basic and diluted share. ... period in 2014, of $8.7 million, or $0.27 per basic ... cash equivalents and investments totaled $117.5 million. , "During ...
(Date:4/30/2015)... Cambridge, MA & Budapest, Hungary (PRWEB) April 30, ... the only web-based electronic lab notebook with integrated ... leader in providing chemistry software solutions and consulting ... that led to the development of Labguru’s newest ... a set of deep domain features for biologists, ...
Breaking Biology Technology:Pfenex To Report First Quarter 2015 Results and Provide Business Update on Thursday, May 14, 2015 2Antibody Production Market worth $2.572 Billion by 2019 2Antibody Production Market worth $2.572 Billion by 2019 3Antibody Production Market worth $2.572 Billion by 2019 4Cytokinetics, Incorporated Reports First Quarter 2015 Financial Results 2Cytokinetics, Incorporated Reports First Quarter 2015 Financial Results 3Cytokinetics, Incorporated Reports First Quarter 2015 Financial Results 4Cytokinetics, Incorporated Reports First Quarter 2015 Financial Results 5Cytokinetics, Incorporated Reports First Quarter 2015 Financial Results 6Cytokinetics, Incorporated Reports First Quarter 2015 Financial Results 7Cytokinetics, Incorporated Reports First Quarter 2015 Financial Results 8Labguru Integrates ChemAxon's Marvin JS, to Enhance Functionality for Chemists 2
... Richard Gabriel, Chief,Executive Officer of DNAPrint Genomics, ... be featured in an exclusive interview with, http://www.wallst.net ... September 10 at 11,a.m. EDT. The interview will ... p.m.,EDT on September 10. The interview will ...
... Alba Therapeutics Corporation today,announced the appointment of ... Clinical Development and Medical Affairs. In this role, ... clinical development strategy,and program execution through product approval ... a member of Alba,s executive management,leadership team. ...
... /PRNewswire-FirstCall/ - ARIUS Research Inc., (TSX:,ARI), a biotechnology ... therapeutics, today announced that it will deliver poster,presentations ... of its anti-cancer antibody drug candidates. At ... on September 7-8,Arius will report recent results of ...
Cached Biology Technology:WallSt.net (www.wallst.net) Announces Upcoming Interview With CEO of DNAPrint Genomics 2Alba Therapeutics Appoints Dr. Betsy Van Parijs as Its Senior Vice President, Clinical Development and Medical Affairs 2Arius Features Preclinical Data on Antibody Candidates at Two Conferences 2Arius Features Preclinical Data on Antibody Candidates at Two Conferences 3
... liter of urine will give a positive reaction ... ,Certification: The Donors are of US origin ... NEGATIVE for HbsAg, HCV, HIV-1 & 2, Syphilis ... THIS MATERIAL IS SOLD FOR IN-VITRO DIAGNOSTIC USE ...
... Identical to the DH-2000, except that it ... 190-1700 nm wavelength range and comes with a ... up to 5 Hz) The DH-2000 ... spectrum of deuterium and tungsten halogen light sources ...
... pump is a dedicated cuff pump for small ... replaces manual inflation and can be used for ... mouse and rat; from single up to multi-channel ... pump has adjustable cut-off pressure in 50 mmHg ...
... These handy dispensers save space ... hats, and other garb. They are ... small parts used in labs, processing ... static charges and the particles they ...
Biology Products: