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.
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.
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.
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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Cystic Fibrosis Foundation, the disease advocacy group that funded the early work on Kalydeco, had always intended to find a way to make the medicine work in patients whose disease was caused by other genes.
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One disease under study is cystic fibrosis, which is caused by one of around 200 defects on a single gene.
But in conditions such as cystic fibrosis or chronic obstructive pulmonary disease (COPD), the "brush" fails to work properly, they suggest, becoming squashed and trapping mucus, which then becomes stuck to cells.
Twenty-two years ago Francis Collins, who would go on to head the Human Genome Project and run the National Institutes of Health, was part of a team that found the gene that, when mutated, caused cystic fibrosis, a deadly childhood disease in which the lungs fill with mucus and the pancreas does not make digestive enzymes.
There are other children born with disease and disability, like cystic fibrosis or juvenile diabetes.
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.
People with mental health problems, or conditions such as asthma, Parkinson's disease, cancer, multiple sclerosis or cystic fibrosis must pay for many of their prescribed medicines and dressings.
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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Compounds in preclinical tests target cystic fibrosis (30, 000 people plus a thousand new cases per year), Huntington's disease (30, 000 cases) and Rett syndrome, a rare inherited brain disorder.
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