In addition, women carrying the genes, it was feared, might be more prone to side-effects such as pain, shortness of breath and fibrosis - the formation of scar-like tissue.
Regulus recently published some very intriguing data with miR-21 in cancer and fibrosis models (the subject of their earlier deal with Sanofi), as well as miR-33 in lipid biology.
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If you have a lot of scarring and fibrosis, the face doesn't work like it should - the eyelids can't close, the nose won't work, and the mouth won't work.
Inflammation and fibrosis - scarring - of the liver tissue, which affects about 10% of the UK population, can cause cirrhosis or liver failure and lead to cancer if diagnosis is delayed or if left untreated.
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Things that can predispose to chronic bacterial infection include allergies, oral infections, an anatomic blockage such as a deviated nasal septum, nasal polyps or enlarged tonsils, frequent swimming, intranasal cocaine use, immunodeficiency syndromes and diseases such as cystic fibrosis and diabetes.
This compound will enter initial clinical trials early next year for skin and lung fibrosis.
He participates in nearly every big-name fundraiser that takes place in Montreal, including those for breast cancer, Lou Gehrig's disease and cystic fibrosis.
Four charities including CP Sport, Hope Against Cancer, Muscular Dystrophy Campaign and Pulmonary Fibrosis Research will benefit from funds raised on the day.
For years, researchers have looked for ways of treating genetic diseases like muscular dystrophy, hemophilia and cystic fibrosis by targeting the genetic defects that cause them.
Children's Hospital at Vanderbilt University, has seen teenage patients with chronic health conditions such as diabetes, congenital heart disease and cystic fibrosis age out of their family's insurance.
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For some inherited disorders such as Huntington's disease and cystic fibrosis, where a change in just a single gene causes a potentially devastating condition, highly predictive genetic tests already available.
Professor Danny McAuley from the Centre for Infection and Immunity, a partner in developing the new nanomedicine, said that, if successful, this nanomedicine could also have application in other common lung disorders such as Chronic Obstructive Pulmonary Disease (COPD) and Cystic Fibrosis.
And two companies, Alnylam Pharmaceuticals and Sirna Therapeutics, are trying to develop RNAi as a drug against diseases like macular degeneration, cystic fibrosis and pandemic flu.
Products based on marine organisms have already found their way onto the market and are now being prescribed to sufferers of asthma, tuberculosis, cancer, Alzheimer's disease, cystic fibrosis and male sexual impotence, among others.
Hayes said his organization supported the use of embryo screening to help prevent the passing on of serious diseases and disorders like Cystic Fibrosis, but is wary of other technologies and how genetic screening and alteration can be misused.
The company has tried to create new enthusiasm with with efforts to expand into hepatitis C, the viral liver disease that is looking like the next big drug market, and into other areas such as heart disease, cystic fibrosis, and pulmonary arterial hypertension.
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The money raised has been split between Royal Manchester Children's Hospital and the Cystic Fibrosis Trust.
She started pilot programs in cystic fibrosis and bronchiolitis to see if doctors would follow stricter medical guidelines.
If a new combination of Kalydeco and another cystic fibrosis pill proves effective, it would likely become a blockbuster.
Screening services for cystic fibrosis and Down's syndrome are to be made available to all new mothers and pregnant women.
Genetic screening, including prenatal screening for cystic fibrosis and other genetic disorders, accounts for a smaller piece of the market.
It can be used to check an embryo does not carry any one of 50 different conditions including cystic fibrosis and Duchenne muscular dystrophy.
Shivers' attorney has described him as an "unlikely terrorist" because he had cystic fibrosis and a doctor had told him he only had a few years to live.
Genes for cystic fibrosis and muscular dystrophy soon followed.
Most was to pay for the development of new medicines for chronic lung diseases such as cystic fibrosis and emphysema, which can, in theory, be made from proteins in the milk of a cloned flock.
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It is urging families and patients with cystic fibrosis, including those with hopes for future drugs in the pipeline, to contact their assembly members and ask them to lobby Mr Drakeford not to accept the recommendation.
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As the source of every cell, tissue and organ in the body, their medical application has the potential to repair damaged tissue, regrow internal organs and cure currently incurable diseases like cystic fibrosis, Alzheimer's and Parkinson's.
Recent improvements in screening and treatments mean most people with cystic fibrosis now live into their 20s and 30s, and some are living into their 40s and 50s.
It has worked phenomenally for organizations like the Cystic Fibrosis Foundation and the Multiple Myeloma Research Foundation.
Not all of these new drugs came from big pharma as biotech companies like Regeneron (Eylea for wet acute macular degeneration), Vertex (Kalydeco for cystic fibrosis) and Incyte (Jakafi for bone marrow disease) had major drugs approved.
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