Navigation Links
FDA OKs Drug That Targets Rare Form of Cystic Fibrosis
Date:1/31/2012

TUESDAY, Jan. 31 (HealthDay News) -- Kalydeco, the first drug that targets the defective protein behind a rare form of the deadly lung disorder cystic fibrosis, was approved Tuesday by the U.S. Food and Drug Administration.

One patient advocate group applauded the decision.

"Today marks an important milestone in our journey to find a cure for cystic fibrosis," Robert Beall, president and CEO of the Cystic Fibrosis Foundation, said in a news release. "Kalydeco addresses the underlying cause of cystic fibrosis, and the science behind the drug has opened exciting new doors to research and development that may eventually lead to additional therapies that will benefit more people."

People with cystic fibrosis have mutations in a gene that produces a protein called CFTR, which regulates the movement of ions (such as chloride) and water in the body. This defect causes sticky mucus to accumulate in the lungs and other organs, resulting in infections, digestive problems and usually death in early adulthood.

Cystic fibrosis affects an estimated 30,000 people in the United States and is the most common fatal genetic disease in the white population. About 1,200 (4 percent) of cystic fibrosis patients have a rare form of the disease caused by a specific G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene, the FDA explained in a news release.

The twice-a-day pill Kalydeco (ivacaftor) was approved for patients with this rare form of cystic fibrosis. The pill, which is taken with fat-containing food, helps the protein made by the CFTR gene function better, the FDA explained.

Two 48-week clinical trials involving 213 patients -- one with patients ages 12 and older and the other with patients ages 6 to 11 -- showed that the drug improved lung function and reduced other symptoms of cystic fibrosis.

The most common side effects of the drug were upper respiratory tract infection, headache, stoma
'/>"/>

Copyright©2010 ScoutNews,LLC.
All rights reserved

Page: 1 2

Related medicine news :

1. New Study: Improved Immune System with Gene-Eden, a Natural Antiviral Supplement that Targets Chronic Viruses
2. Hot Pepper Itch Remedy Targets Kidney Disease Patients Suffering From Chronic Pruritus
3. Scott & White Healthcare researchers studying investigational agent that targets breast cancer
4. Global Leader Readied For Healthy Aging Boom, Targets Wall Street With New Center In Trump Building
5. FDA Targets Safety Problems With Infusion Drug Pumps
6. Global Summit on International Breast Health Targets Breast Health Improvements in Developing Countries, June 9-11, 2010, in Chicago
7. Combination therapy targets stubborn leukemia stem cells
8. Miami meeting on radiation therapy targets patient safety
9. Researcher targets gene regulators on link between arsenic, cancer
10. MSU targets womens health research with $2.5 million grant
11. TGen finds therapeutic targets for rare cancer in children
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/19/2014)... illness-causing E. coli bacteria were found on nearly one-quarter ... new study. Researchers checked cilantro, basil and parsley ... Los Angeles and Orange counties in California, and in ... 24 percent were positive for E. coli. One sample ... Both types of bacteria can cause sometimes serious or ...
(Date:12/19/2014)... The cost of cancer treatment can cause financial stress ... Researchers examined data from 1,000 people in the United ... cancer. Of those patients, almost 900 had finished their ... advanced cancer. Nearly half of the patients said ... was linked with a poorer health-related quality of life. ...
(Date:12/19/2014)... Diet Doc strives to continue to provide ... weight loss by combining scientific fast weight loss ... company understand that remaining committed to a diet can ... and sugars from the diet can cause patients to ... loss goals. , For this reason, Diet Doc ...
(Date:12/19/2014)... December 19, 2014 B. E. Smith, ... healthcare providers, has been retained to lead a national ... Hospital in Franklin, Ind. The top executive search firm ... placed more than 900 healthcare executives into organizations. ... service hospital providing care to the residents of Johnson ...
(Date:12/19/2014)... Mobile Spa, has announced they now have a Virtual ... into a utopia for the Long Island community where the ... Tour showcases the beauty of the Spa On Wheels which ... The Virtual Tour shows viewers each of the rooms in ... Marla Kaplan-Pelle, Director states " It's hard to imagine that ...
Breaking Medicine News(10 mins):Health News:E. Coli Germs Found on Farmers Market Herbs 2Health News:Cancer Treatment Costs Weigh Heavily on Patients, Study Finds 2Health News:Diet Doc Incorporates Appetite Zap into their Already Impressive Collection of Diet Products to Jump Start the Metabolism and Burn Fat 4X Faster 2Health News:Diet Doc Incorporates Appetite Zap into their Already Impressive Collection of Diet Products to Jump Start the Metabolism and Burn Fat 4X Faster 3Health News:Diet Doc Incorporates Appetite Zap into their Already Impressive Collection of Diet Products to Jump Start the Metabolism and Burn Fat 4X Faster 4Health News:Johnson Memorial Hospital Retains B. E. Smith to Recruit New Chief Financial Officer 2Health News:Johnson Memorial Hospital Retains B. E. Smith to Recruit New Chief Financial Officer 3Health News:A Virtual Tour Of Hands On Mobile Massage Spa Is Now Available 2Health News:A Virtual Tour Of Hands On Mobile Massage Spa Is Now Available 3
... greatly increases the risk of heart diseases as an adult, according ... heart disease is not how much you weigh now, but how ... with a low birth weight are roughly twice as susceptible to ... an epidemologist at the University of Southampton, and a team at ...
... The right side of the brain helps people recognise ... from the Beth Israel Deaconess Medical Center in Boston ... ,The study joins a growing body of evidence ... in self-awareness.// Studies of self-awareness may provide unique insights ...
... who do not express their requests feel less satisfied with their ... not tell a patient everything on his mind. One may feel ... she may seem rushed, or it may be that the relationship ... as it could be.// ,Researchers at the University of ...
... differences in disease patterns in different mammals - and it ... genes with our primate cousins - yet they rarely get ... dogs do not suffer from high blood pressure, even when ... Professor of Comparative Medicine at the University of Houston and ...
... tool to the field of medicine. An interactive video ... treatment. The days when doctors told you what treatment ... participate in the decision-making process.// But sometimes this is ... new research comes up all the time. ...
... or sleep apnoea has the potential to alter voice characteristics. ... stops many times during the night - is to remodel ... is that the uvula - a flap of tissue at ... ,If the uvula is trimmed - in an operation called ...
Cached Medicine News:Health News:The right is responsible 2
(Date:12/19/2014)... , Dec. 18, 2014 Somewhere between dropping ... health was forgotten. But Audicus , a next-generation ... better this new year. Hearing loss is ... aging population, though it is often unaddressed. Forty-eight million ... has a hearing aid, mainly due to price. Hearing ...
(Date:12/19/2014)... 18, 2014  RESMED INC. (NYSE: RMD ) ... fiscal year 2015 results on Thursday, January 22, 2015, after ... with ResMed,s results will be issued after 1:00 p.m. US ... discuss operating results and future outlook. The earnings ... Time and the live webcast of the call can be ...
(Date:12/19/2014)... has witnessed robust development with the growth pace keeping at ... In 2012, the size of China ... worldwide soaring to 16.3%. It is projected that ... rate by around 20% in the upcoming years and that ... An integrated membrane industrial system has taken shape ...
Breaking Medicine Technology:Audicus Will Help People Resolve to Hear Better in 2015 2ResMed To Announce Second Quarter 2015 Results 2Separation Membranes Market (Company, Country & Application) Research Reports for Global and Chinese Regions 2Separation Membranes Market (Company, Country & Application) Research Reports for Global and Chinese Regions 3
... a system that measures the sedimentation ... rate may be relevant as input ... rate is measured in a batch ... in high-volume settings such as hospitals ...
... of automatic systems for performing erythrocyte sedimentation ... blood. With the introduction of the Ves-matic ... market a truly revolutionary testing system which ... with the traditional ESR techique.,By means of ...
... The NEW VACUETTE® erythrocyte ... user-friendly, can help. The new generation ... existing analysers with whole range of ... analysis of the erythrocyte sedimentation rate. ...
The MICROsed-System is the ideal system back up support to be used as front-line instrument with small workloads....
Medicine Products: