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Tag Archives: cystic-fibrosis-
NHS in England Funds KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis, for …
CAMBRIDGE, Mass.–(BUSINESS WIRE)– Vertex Pharmaceuticals Incorporated (VRTX) announced today that a decision has been made by the National Health Service (NHS) in England to fund KALYDECO (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF), for people ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Cystic fibrosis is a rare genetic disease for which there is no cure. It is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Continue reading
Producing Viral Vectors for Clinical Trials With On-Site Clinical Manufacturing Facility – Video
Producing Viral Vectors for Clinical Trials With On-Site Clinical Manufacturing Facility Viral gene therapy is being tested as treatment for numerous diseases, including cancer, cystic fibrosis, heart disease, muscle diseases, diabetes and neurodegenerative disease like Parkinson's. Continue reading
A genetic counsellor share’s the job’s highs and lows
Genetic counsellors guide people and families through the complicated nature of a genetic conditions such as cystic fibrosis, cancer and mental illness. The Star spoke with Riyana Babul-Hirji, a genetic counsellor at Sick Kids Hospital, about the job. Q: Whatdoes a genetic counsellor do? Continue reading
New Jersey Medical School Researchers Develop DNA Sequencing Tests for Hereditary Diseases
Newswise NEWARKScientists at the University of Medicine and Dentistry of New Jersey-New Jersey Medical School (UMDNJ-NJMS) have developed new DNA sequencing tests that hold significant promise for decreasing costs associated with diagnosing cancer and hereditary diseases, including cysticfibrosis. Officials at the New Jersey Department of Health approved the use of the new Cystic Fibrosis (CF) Carrier and Diagnosis Test, which was created at the Institute for Genomic Medicine at UMDNJ-NJMS. Using a semiconductor mechanism that was developed by San Francisco-based Ion Torrent, the microchip tests the entire gene for mutations Continue reading
3 Health-Care Stocks Crushing the Market
May isn’t in the books yet, but three health-care stocks have shot to the stratosphere. Unsurprisingly, the month’s top gainers are all from the biotech space, but not all are small-cap companies. Let’s take a moment to reflect on their dramatic moves, and which one has the most room to keep running, over the holiday weekend Continue reading
Emory medical school gets $5 million gift
ATLANTA (AP) – The Emory University School of Medicine has received a commitment for a $5 million gift for its pediatrics department. The money from the Marcus Foundation, Inc., will be used to create the Marcus Society in Pediatrics. Continue reading
Gene Therapy in Detail – Video
09-02-2012 05:43 Dr Mat Hickman explains gene therapy as a possible treatment for Cystic Fibrosis to Natasha, one of the Changing Futures participants. Continue reading
What is Gene Therapy – Animation – Video
12-03-2012 05:50 This clever little animation explains the process of gene therapy for Cystic Fibrosis … and it was designed by Changing Futures participant Caitlin. This film is part of the Changing Futures Project: www.changing-futures.co.uk. Continue reading
What is Gene Therapy (The General Explains) – Video
12-03-2012 05:53 This clever little animation explains the process of gene therapy for Cystic Fibrosis … and it was designed by Changing Futures participant Caitlin. Continue reading