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Help Children With Rare Diseases Get the New Medicines They Need
July 5, 2011
RemedyMD and the Children’s Rare Disease Network Announce Plan to Give Away Free Rare Disease Research Registry Valued up to $50,000 via Contest
February 25, 2011
7,000 Bracelets for Hope™ Campaign Raises Awareness For Over 7000 Rare Diseases
February 08, 2011
Global Genes Project Announces "Wear That You Care!™" Denim Awareness Camaign for Rare Disease Day 2011
January 26, 2011
Thumbelina Kids: Tiny as Dolls, They Strive to Fit In
January 18, 2011
2010 ROCK STARS OF SCIENCE!" DR. EMIL KAKKIS HONORED AS A 2010 ROCK STAR OF SCIENCE
November 17, 2010
7,000 Bracelets For Hope™ - A Rare Disease Awareness Campaign
November 1, 2010
Global Genes Quarterly Conference Call Overview
October 28, 2010
Seeking A Cure For Jonah
October 19, 2010
The Global Genes Project Charity Benefit and Fashion Week Kick Off!
September 7, 2010
Advocates to bring rare disease philanthropy under one umbrella
August 9, 2010
Big pharma moves from 'blockbusters' to 'niche busters'
August 9, 2010
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
Children's Rare Disease Network Partners With Medpedia.com To Create Rarespace
June 29, 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
Pfizer's Viagra Faces FDA Review for Use in Children With Lung Condition
By Shannon Pettypiece and Molly Peterson - Jul 27, 2010
A form of Pfizer Inc.’s erection drug Viagra, sold as the blood-pressure treatment called Revatio in adults, may be used for children with a rare lung disorder if U.S. regulators can agree on how to test it.
The condition, called pulmonary arterial hypertension, affects only 600 children a year, New York-based Pfizer said. It causes high blood pressure in arteries in the lungs, making the right side of the heart work harder than normal and causing chest pain, dizziness and fatigue. Outside advisers to the Food and Drug Administration are set to meet July 29 to evaluate whether Pfizer’s study of Revatio is sufficient to determine its effectiveness in children, the agency said today.
If the New York-based company meets FDA requirements, the drug would get an extra six months on the market without generic competition. Patents on the drug, with 2009 sales of $1.89 billion as Viagra and $450 million as Revatio, are expected to expire in 2012. Some doctors are already using the treatment in kids with the lung condition.
“It’s a good option in pediatric patients because it is well-tolerated, in that it doesn’t have as many side effects as some of the other options,” said Chad Knoderer, a pediatric clinical pharmacist at Riley Hospital for Children in Indianapolis, who has used Viagra in kids with the disorder.
FDA Request
The FDA in 2001 asked Pfizer, the world’s biggest drugmaker, to study the medicine in children with the lung disorder. Sildenafil, the chemical name for both Viagra and Revatio, blocks an enzyme found in the lungs and penis that regulates blood flow. Pfizer is considering whether to seek approval for Revatio, a lower-dose form of sildenafil, in children, said Colin Ewen, an executive director at the company, in a July 23 telephone interview.
Pfizer shares rose 16 cents, or 1.1 percent, to $15.18 at 10:09 a.m. in New York Stock Exchange composite trading.
The FDA is asking its panel of advisers to decide what is the best measurement to use in determining if the drug is effective in children with pulmonary hypertension.
In adults, researchers typically use an exercise test to determine if the drug is having a benefit. Because that test was difficult to perform in young children, Pfizer has asked the FDA to consider an alternative test that measures blood flow by inserting a catheter through the arteries.
In Pfizer’s study of 234 children, the drug failed to show a benefit when the children’s exercise ability was measured. However, it did show a significant benefit when researchers used an alternative measure of blood flow.
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