Sentences with phrase «cystic fibrosis drugs»

«Unsuspected characteristics of new cystic fibrosis drugs found, offering potential paths to more effective therapies.»

«New imaging technique shows effectiveness of cystic fibrosis drug: Hyperpolarized helium MRI could aid in development of improved therapies for lung diseases.»

Not exact matches

'» (Translate is planning human trials with repeated doses of its own mRNA drug for both cystic fibrosis and a rare metabolic disorder called ornithine transcarbamylase deficiency in 2018, but it has not yet published animal studies with repeat dosing.)

People with an uncommon form of cystic fibrosis started gaining weight and were better able to breathe than their untreated counterparts after just two weeks on an experimental drug, according to a study published November 2 in The New England Journal of Medicine.

A study published by scientists at University of Massachusetts Medical School and the University of Alabama at Birmingham provides insight into the mechanism of action of the drug ataluren, which is showing promise in treating Duchenne muscular dystrophy and cystic fibrosis.

Appearing alongside the heads of five of NIH's 27 institutes and centers, Collins instead offered examples of how NIH research has led to new drugs for cystic fibrosis and cancer treatments that help the immune system fight tumors.

In lab experiments using tissue samples cultured from cystic fibrosis patients, scientists at the UNC School of Medicine and the UNC Marsico Lung Institute have shown that a new CF drug counteracts the intended beneficial molecular effect of another CF drug.

«The molecular architecture of this membrane protein is crucial for the targeted development of drugs for treating cystic fibrosis,» emphasizes Dutzler.

If a drug works, the complete cystic fibrosis protein will be made — and scientists will see a glow.

In the Netherlands, based on research reported in 2016 in Science Translational Medicine, Clevers and colleagues are already using personalized gut organoids, derived from rectal biopsies, to test whether cystic fibrosis patients will benefit from available drugs.

To screen for such drugs, scientists attach firefly luciferase DNA to the end of the cystic fibrosis gene.

His team looked at an experimental drug called PTC124, which the firefly luciferase assay had identified as a promising candidate for treating stop - sign forms of cystic fibrosis and which is now being tested in clinical trials.

«Because airway spheres from a patient with cystic fibrosis do not swell in our assay but airway spheres from a healthy person do, we can see whether adding a certain drug or combination of drugs causes them to swell more.

Cystic fibrosis patient Els van der Heijden received a new drug combination based on organoid tests.

Fast screening — coupled with $ 75 million from the Cystic Fibrosis Foundation — allowed Vertex of Cambridge, Massachusetts, to develop Kalydeco and two other drugs being tested.

It is a landmark moment: 22 years after the cystic fibrosis gene was discovered, some patients are about to receive a drug called Kalydeco to treat the defect that causes their lungs to clog up with sticky mucus.

The decision is expected to delay the provision of nine other new treatments and services by NHS England, including prosthetics for lower limb loss, hearing implants for children with auditory nerve problems, and drugs for some children with cystic fibrosis.

A DRUG has improved lung function in children with cystic fibrosis, raising hopes that the life - threatening damage caused by the genetic disease can be halted or even reversed.

Kalydeco is already helping hundreds of people, according to Robert Beall, president of the Cystic Fibrosis Foundation: «The drug opens new doors that may help us find therapies for everyone with this terrible disease.»

Since the gene behind cystic fibrosis was discovered in 1989, researchers have tried to develop drugs that directly target the faulty protein it makes in those with the disease.

Urged by desperate parents, the board expanded the traditional nonprofit by launching Cystic Fibrosis Foundation Therapeutics Inc., an independent arm, to spearhead drug discovery.

By combining Kalydeco with another drug to create a single medicine called Orkambi, clinicians have since attempted to extend treatment to half of all people with cystic fibrosis.

«More drugs are under development to treat cystic fibrosis and other lung conditions, and improved imaging techniques are needed to test their effectiveness,» Altes said.

«Major cystic fibrosis breakthrough made: Drug combination improves lung function.»

Ivacaftor is the first drug to treat the underlying causes of cystic fibrosis rather than just its symptoms and is currently approved for patients with the «celtic gene» mutation carried by about four per cent of all patients and 10 - 15 per cent of patients in Ireland.

«Potential new drug target for cystic fibrosis.»

Scientists at the European Molecular Biology Laboratory (EMBL) in Heidelberg and Regensburg University, both in Germany, and the University of Lisboa, in Portugal, have discovered a promising potential drug target for cystic fibrosis.

Quirk in firefly - enzyme - based assay may throw into question the discovery of drugs for cystic fibrosis and other conditions

For example, organoids grown from the guts of individual cystic fibrosis patients are used to predict their response to drugs.

We are going to screen a larger library of chemicals to identify molecules that either boost or weaken NMD, which should help develop better and more targeted drugs for treating ALS, muscular dystrophy and cystic fibrosis.»

They have also been used advantageously for personalized mutation and drug screening in cancer and hereditary diseases such as cystic fibrosis, and for studying infection or inflammatory diseases involving host - pathogen interactions (2,7 - 12).

Overlap — Finally, successful drug targets significantly overlap with disease genes — those in which a mutation can cause a specific disease, such as cystic fibrosis — and with essential genes, that are required for normal development.

Protein function discoveries in the past 20 years have been the impetus for therapies for cystic fibrosis, and the ability to inhibit certain types of protein expression is at the heart of cholesterol - lowering statin drugs.

Cystic Fibrosis Foundation Therapeutics Established in 2000, Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) is the nonprofit drug discovery and development affiliate of CFF.

Novel drug therapies and novel indications of drug therapy, e.g. tyrosine kinase inhibitors in lung disease other than lung cancer, biological treatments for asthma, COPD, cystic fibrosis and interstitial lung disease.

Now that we understand why, we can design better drugs to combat cystic fibrosis in humans.»

A common antibacterial substance found in toothpaste may combat life - threatening diseases such as cystic fibrosis, or CF, when combined with an already FDA - approved drug.

The mission of the Cystic Fibrosis Foundation is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high - quality, specializedCystic Fibrosis Foundation is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high - quality, specializFibrosis Foundation is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high - quality, specializedcystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high - quality, specializfibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high - quality, specialized care.

The Canadian Cystic Fibrosis Foundation ended 2010 with one of the year's more interesting collaborations between nonprofit organizations and commercial drug developers.

The mission of the Cystic Fibrosis Foundation is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high - quality, specializedCystic Fibrosis Foundation is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high - quality, specializFibrosis Foundation is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high - quality, specializedcystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high - quality, specializfibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high - quality, specialized care.