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
Tag Archives: a-rare-genetic
Gene Therapy on the Mend
The first regulatory approval of a gene therapy treatment could spark broader patient access to the technology. Continue reading
Gene Therapy on the Mend as Treatment Gets Western Approval
Regulatory approval of a gene therapy treatment in Europe could spark broader patient access to the technology. Continue reading
Europe approves high-price gene therapy
LONDON (Reuters) – European officials have approved the Western world’s first gene therapy drug from a small Dutch biotech company, in a milestone for the novel medical technology that fixes faulty genes. The formal clearance from the European Commission paves the way for a launch next summer of the treatment for an ultra rare genetic disease that will cost around 1.2 million euros ($1.6 million) per patient, a new record for pricey modern medicines Continue reading
Europe nears gene therapy first
20 July 2012 Last updated at 11:11 ET By James Gallagher Health and science reporter, BBC News Europe is on the cusp of approving a gene therapy for the first time, in what would be a landmark moment for the field. Gene therapies alter a patient’s DNA to treat inherited diseases passed from parent to child Continue reading
Breaking the skin barrier: Drugs topically deliver gene therapy via commercial moisturizers for skin disease treatment
ScienceDaily (July 2, 2012) Getting under your skin takes on a brave new meaning thanks to Northwestern University research that could transform gene regulation. Continue reading
Moffitt Cancer Center Study Validates Activity of Rare Genetic Variant in Glioma
Newswise Researchers at Moffitt Cancer Center working with colleagues at three other institutions have validated a link between a rare genetic variant and the risk of glioma, the most common and lethal type of brain tumor. The validation study also uncovered an association between the same rare genetic variant and improved rates of survival for patients with glioma. Continue reading
My Life. My Medicine. – Luke’s Story – Video
28-06-2012 02:51 Luke is an eighteen-year-old medical marijuana patient in California. He has been suffering from a rare genetic skin disorder his whole life that causes his skin can to blister and tear with just slight contact. Continue reading
Parents must be considered during expansion of genetic screening programs for newborns
Parents must be considered when states decide to expand genetic screening programs for newborns, according to a new study that looked at mandatory testing panels and political pressure by advocacy groups. Continue reading
Gene Therapy Helps Treat Children with Rare Brain Disorder
Gene therapy provides new hope for children with AADC deficiency, a rare genetic disorder that causes neurological impairment and impacts a child's ability to move and eat. Continue reading
Family hangs hope for boy on unproven therapy in India
Indian clinic’s stem cell therapy real? STORY HIGHLIGHTS For more of CNN correspondent Drew Griffin’s investigation of India’s experimental embryonic stem cell therapy, watch “CNN Presents: Selling a Miracle,” at 8 and 11 p.m. ET Sunday on CNN. Continue reading