PRESS
Pfizer's Viagra Faces FDA Review for Use in Children With Lung Condition
July 27, 2010
NIH Takes On New Role in Fight Against Rare Diseases
July 24, 2010
NORD Testifies Before Senate HELP Committee
July 21, 2010
A Great Win for Rare Diseases in U.S. Senate Appropriation Bill
July 15, 2010
Regulatory Flexibility
July 02, 2010
FDA Database Aims to Spark Orphan-Disease Drug Development
June 18, 2010
EXCLUSIVE: Pfizer plans to move fast on rare disease pacts
June 17, 2010
Good news for rare disease?
June 15, 2010
THE CHILDREN'S RARE DISEASE NETWORK LAUNCHES VALUABLE INFORMATIONAL BLOG
June 9, 2010
FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease
May 17, 2010
Genetic Sequencing Kit Catches Rare Mutation for TARP Syndrome
May 15, 2010
Parents of child with rare illness aim to help
April 26, 2010
AltheaDx and The Nicholas Conor Institute for Pediatric Cancer Research Announce Molecular Diagnostics Collaboration to Improve the Diagnosis and Treatment of Childhood Cancer
April 19, 2010
Cooking with the Genzyme Recipe: New Players Funding Rare Disease Drugs in Boston
April 12, 2010
PhRMA Honors Patient Advocates Ron and Raychel Bartek
March 18, 2010
A Legacy For and Beyond Batten Disease
March 16, 2010
Study opens new avenue for developing treatments for genetic muscle-wasting disease
March 15, 2010
Novato's BioMarin finds niche and growing quickly
March 13, 2010
First whole genome sequencing of family of 4 reveals new genetic power
March 10, 2010
Push to Cure Rare Diseases
March 10, 2010
NIH-Funded Research Study
March 8, 2010
250 Million People Worldwide Estimated to Suffer From Rare Disease
March 8, 2010
GENE THERAPY REVERSES EFFECTS OF LETHAL CHILDHOOD MUSCLE DISORDER IN MICE
February 28, 2010
CHI SUPPORTS RESEARCH AND HOPE FOR PATIENTS OF RARE DISEASES
February 25, 2010
RARE DISEASE ADVOCATES UNITE TO TRANSLATE SLOGAN OF GLOBAL GENES PROJECT IN TIME FOR WORLD RARE DISEASE DAY!
February 25, 2010
reco® jeans SUPPORTS CHILDREN WITH RARE DISEASES
February 23, 2010
MILLIONS AROUND WORLD TO OBSERVE RARE DISEASE DAY ON SUNDAY
February 23, 2010
GLOBAL GENES PROJECT TO RAISE AWARENESS FOR MILLIONS OF CHILDREN LIVING WITH RARE DISEASE
February 1, 2010
GALAPAGOS TO FOCUS ON RARE DISEASES IN STRATEGIC SHIFT
January 26, 2010
THE PATIENT ASCENDANT
January 18, 2010
FUTURE OF NEWBORN SCREENING ENVISIONED: PROCEEDINGS NOW VIEWABLE ONLINE
January 7, 2010
HUNTING NEWBORN TESTS FOR SUPER-RARE GENE DISEASES
January 5, 2010
THE LONELINESS OF FIGHTING A RARE CANCER
January 5, 2010
DONATE GAMES CHARITY CONNECTS COMMUNITIES WORLDWIDE
December 21, 2009
DONATEGAMES TURNS USED VIDEO-GAMES INTO FUNDING FOR MEDICAL RESEARCH TO HELP KIDS
November 25, 2009
CHILDREN'S RARE DISEASE NETWORK RECEIVES LIFE TECHNOLOGIES FOUNDATION GRANT
November 17, 2009
BABY Z CURED OR RARE DISEASE IN 3 DAYS
November 11, 2009
SOCIAL NETWORKING SAVIORS: TWITTER, FACEBOOK USED IN EFFORT TO HELP SAVE A BABY'S LIFE
October 29, 2009
U.S. AND EUROPEAN RARE DISEASE ORGANIZATIONS SIGN STRATEGIC ALLIANCE
October 28, 2009
RARE DISEASE CENTER HOSTS SYMPOSIUM ON NEW STRATEGIES
October 27, 2009
RARE FIND
October 23, 2009
NEW FDA GROUPS FOR RARE, NEGLECTED DISEASES COULD SPEED PATH TO MARKET
October 12, 2009
ARNOLD NATIVE TO RUN ACROSS SAHARA DESERT
August 18, 2009
CAMP SUNDOWN SHINES IN THE BRONX
August 13, 2009
RESEARCHERS IDENTIFY NEW FUNCTION FOR PROTEIN MISSING IN DUCHENNE MUSCULAR DYSTROPHY
August 4, 2009
AMERICANS STRUGGLE TO PAY FOR HEALTHCARE: STUDY
June 22, 2009
DEAL REACHED TO CUT DRUG COSTS
June 20, 2009
ONE GIRL'S HOPE, A NATION'S DILEMMA
June 14, 2009
IN RARE DISEASE, A FAMILIAR PROTEIN DISRUPTS GENE FUNCTION
May 26, 2009
NEW INSTITUTE WILL STUDY RARE DISEASE
May 20, 2009
UNC-DUKE STUDY: IMPAIRED BRAIN PLASTICITY LINKED TO ANGELMAN SYNDROME LEARNING DEFICITS
May 10, 2009
TO MAKE PROGRESS IN RARE CANCERS, PATIENTS MUST LEAD THE WAY
May 9, 2009
MO. VOTES TO ADD 5 DISEASES TO NEWBORN SCREENINGS
May 9, 2009
SIGNATURE GENOMIC LABORATORIES DETECTS CHROMOSOME ABNORMALITIES IN INDIVIDUALS WITH PALLISTER-KILLIAN SYNDROME WITHOUT INVASIVE SKIN BIOPSY
May 7, 2009
MIRACLE FOR MATTHEW
May 5, 2009
SHRINKING BABY MAGGIE AGNEW BAFFLES DOCTORS
May 4, 2009
NEW INSTITUTE WILL STUDY RARE DISEASE
May 20, 2009
WASHINGTON (Reuters) - A unique new institute will look for ways to treat rare and neglected diseases and take the first and riskiest steps toward bringing new drugs to market, U.S. health officials said on Wednesday.
Congress has provided $24 million a year for five years to start the Therapeutics for Rare and Neglected Diseases Program, or TRND at the National Institutes of Health, acting NIH director Dr. Raynard Kington told reporters in a telephone briefing.
The program will use taxpayer money to get drugs through the most costly and dangerous phase of development, known as the "Valley of Death" because so many fail there.
It will publish details of failures as well as successes to guide other researchers, the NIH said.
"Twenty-five to 30 million Americans suffer from rare or neglected diseases," Kington said.
A rare disease is one that affects fewer than 200,000 Americans, and NIH estimates there are about 6,800 of these conditions, ranging from multiple symmetric lipomatosis or Madelung's disease, characterized by large fat deposits around the neck and nervous system abnormalities, to pseudomyxoma peritonei, in which tumor cells swell up the abdomen.
Only about 200 of these conditions, many of which affect fewer than a dozen people, have treatments.
"We don't know yet exactly which diseases this program will take on," Dr. Alan Guttmacher, acting director of the National Human Genome Research Institute, told the briefing.
He said the new institute would be opportunistic, pouncing on promising research studies, some of which may be funded by advocacy groups for rare diseases.
Often drug companies are afraid to take on this work, Guttmacher added. "Getting a promising chemical through the pre-clinical stages of drug development is fraught with failure," Guttmacher said.
"It is colloquially called the "Valley of Death." This stage of drug development can take two to four years of work, costs tens of millions of dollars," and still fail, he added.
90 PERCENT FAILURE
The NIH estimates that up to 90 percent of all potential drugs fail to make it from the lab into human volunteers for safety testing.
The group will publish details even of failures -- something that rarely happens in the world of medical publishing now and a focus that can sometimes lead researchers to cover up or minimize dangers.
"We are going to tell everyone what we are doing," said Dr. Christopher Austin of the NIH Chemical Genomics Center. "That alone will be revolutionary." Early-stage research is often considered proprietary by companies.
One project that may get funding -- a potential new drug to treat schistosomiasis, which is not rare but is considered a "neglected" tropical disease. The parasite kills 280,000 people a year.
A researcher came to the NIH to test a promising new drug and the team published a study last year showing the compound -- still known only by its chemical name 4-phenyl-1,2,5-oxadiazole-3-carbonitrile-2-oxide -- may work.
"When we go to this point there were no resources to carry this project forward. This, I think, is one of the projects that we are going to put into the trend queue," Guttmacher said.
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