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Vertex Pharma (VRTX) Receives Approval for SYMDEKO in Australia to Treat Underlying Cause of CF

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Therapeutic Goods Administration (TGA) of Australia has granted registration to SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) for the treatment of people with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence. Mutations in the CFTR gene that produce CFTR protein responsive to SYMDEKO® include F508del and mutations in which the CFTR protein shows residual function: P67L, R117C, L206W, R3

Gene Therapy

Cystic fibrosis (CF) is a rare, heritable disease caused by mutations in the CFTR gene, which provides instructions for making the CFTR protein. This protein normally forms a channel at the membrane of cells for the transport of salts in and out of the cell. In CF, this channel is either not present, or fails to open because of mutations, which leads to the buildup of sticky mucus in different organs and tissues. While there is currently no cure for CF, there are treatments that can improve patients’ quality of life. Many experimental treatments are also under development. What is gene therapy? Gene therapy is an approach that is intended to replace a faulty gene with a healthy one. To make

Genetic Counseling

Cystic fibrosis (CF) is a genetic disease caused by mutations in the CFTRgene resulting in the buildup of thick, sticky mucus in various organs, such as the lungs, pancreas, intestines, and reproductive organs. The accumulation of this mucus often leads to inflammation and scarring (fibrosis) in the affected organs. Symptoms of the disease vary depending on the affected organ, but most symptoms start showing up soon after birth. There is currently no known cure for CF but there are treatments that can alleviate symptoms. Therapies addressing the underlying cause of the disease have also been developed in recent years. CF is inherited in an autosomal recessive manner. This means that a person

Lower Oxygen Levels During Exercise Is a Risk Factor for Osteoporosis in Non-CF Bronchiectasis, Stud

Decreased blood oxygen levels during the six-minute walk test (6MWT) is a risk factor for osteoporosis in patients with non-cystic fibrosis (CF) bronchiectasis, according to a study. The findings also revealed that having a low body mass index (BMI), advanced age, and severe exacerbations also predicted osteoporosis in this patient population. The research, “Oxygen desaturation during the 6-min walk test as a risk for osteoporosis in non-cystic fibrosis bronchiectasis,” was published in the journal BMC Pulmonary Medicine. Patients with non-CF bronchiectasis experience progressive deterioration of their pulmonary function, and reduced exercise capacity. Osteoporosis — a condition characterize

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