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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