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The Cystic Fibrosis Trust (stylised as Cystic Fibrosis) is a UK-based national charity founded in 1964, dealing with all aspects of cystic fibrosis (CF). It funds research to treat and cure CF and aims to ensure appropriate clinical care and support for people with cystic fibrosis.
Its objectives are:
The aim of the Cystic Fibrosis Trust research is to understand, treat and cure cystic fibrosis. The Cystic Fibrosis Trust is the major funder of medical and scientific CF research in the UK. The Trust's research falls into two main categories:
Cystic fibrosis is a complex disease requiring considerable specialist treatment. In the last fifteen years, the Trust has helped to set up and staff 45 specialist CF treatment centres throughout the UK. Since 1997, the Cystic Fibrosis Trust has invested over £10 million in the NHS to improve clinical care for the 8,000 people in the UK with CF; helping fund doctors, nurses and multidisciplinary teams.
The Cystic Fibrosis Trust sets the national standard on clinical care; provides and funds a UK CF Clinical Database; and measures levels of service provision. The Trust's Expert Patient Advisers (who all have CF) work with health providers and government to influence and improve the care of those with CF across the UK.
The Cystic Fibrosis Trust provides a confidential, 9–5 Monday to Friday helpline service for advice and support on all aspects of cystic fibrosis. The Trust also offers information and advice to those affected, along with their families and friends, schools and employers and anyone interested in cystic fibrosis. The Trust also advise families on benefits and, where appropriate, provides financial assistance and welfare grants.
The Cystic Fibrosis Trust receives donations and support from local communities. [2] In addition to the contributions, fundraising efforts have been undertaken by UK artist Jenny Wicks in 2009 with her photographic art exhibition, short documentary and book titled Root Ginger . The exhibition describes the recessive gene inheritance pattern that causes ginger, or red, hair – the same inheritance pattern that causes cystic fibrosis. [3] A portion of the proceeds from the exhibition and book sales will go to the Cystic Fibrosis Trust.
Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.
Cystic fibrosis (CF) is a genetic disorder inherited in an autosomal recessive manner that impairs the normal clearance of mucus from the lungs, which facilitates the colonization and infection of the lungs by bacteria, notably Staphylococcus aureus. CF is a rare genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. The hallmark feature of CF is the accumulation of thick mucus in different organs. Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. Other signs and symptoms may include sinus infections, poor growth, fatty stool, clubbing of the fingers and toes, and infertility in most males. Different people may have different degrees of symptoms.
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) is one of the institutes and centers that make up the National Institutes of Health, an agency of the United States Department of Health and Human Services (HHS).
Cystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and anion channel in vertebrates that is encoded by the CFTR gene.
Vertex Pharmaceuticals Incorporated is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maintains headquarters in South Boston, Massachusetts, and three research facilities, in San Diego, California, and Milton Park, Oxfordshire, England.
Dorothy Hansine Andersen was the American physician and researcher who first identified and named cystic fibrosis. During her almost thirty year tenure at Babies Hospital of Columbia-Presbyterian Medical Center, Andersen not only identified CF and its inheritance through a recessive gene, she was also at the forefront of developing diagnostic tests and life-extending treatments for the disease. Andersen was also active in researching other diseases that are diagnosed in children. She was the first to describe Glycogen storage disease type IV, which, in recognition of her contributions, became known as Andersen's Disease. Her research on heart malformations informed the development of open heart surgery and the training of new surgeons.
The Cystic Fibrosis Foundation (CFF) is a 501(c)(3) non-profit organization in the United States established to provide the means to cure cystic fibrosis (CF) and ensure that those living with CF live long and productive lives. The Foundation provides information about cystic fibrosis and finances CF research that aims to improve the quality of life for people with the disease. The Foundation also engages in legislative lobbying for cystic fibrosis.
Venture philanthropy is a type of impact investment that takes concepts and techniques from venture capital finance and business management and applies them to achieving philanthropic goals. The term was first used in 1969 by John D. Rockefeller III to describe an imaginative and risk-taking approach to philanthropy that may be undertaken by charitable organizations.
Jeans for Genes Day is a national fundraiser held in Australia and the United Kingdom. The two fundraisers are not associated with one another and raise money for different organisations.
The mission of the Foundation Fighting Blindness is to fund research that will lead to the prevention, treatment and cures for the entire spectrum of retinal degenerative diseases, including retinitis pigmentosa, macular degeneration, Usher syndrome, Stargardt disease and related conditions. These diseases, which affect more than 10 million Americans and millions more throughout the world, often lead to severe vision loss or complete blindness.
Cystic Fibrosis Canada is one of national charitable but not-for-profit corporation established in 1960. Cystic Fibrosis Canada's mandate is to help individuals with cystic fibrosis, principally by funding cystic fibrosis research and care. The organization also provides educational materials for the cystic fibrosis community and the general public; undertakes advocacy initiatives with, and on behalf of Canadians with cystic fibrosis, and raises funds to support its programs.
Ivacaftor is a medication used to treat cystic fibrosis in people with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, who account for 4–5% cases of cystic fibrosis. It is also included in combination medications, lumacaftor/ivacaftor, tezacaftor/ivacaftor, and elexacaftor/tezacaftor/ivacaftor which are used to treat people with cystic fibrosis.
Physiotherapists treating patients following uncomplicated coronary artery bypass surgery surgery continue to use interventions such as deep breathing exercises that are not supported by best available evidence. Standardised guidelines may be required to better match clinical practice with current literature.
Emily's Entourage is a nonprofit organization that raises money and awareness to help find a cure for rare ("nonsense") mutations of cystic fibrosis (CF), a genetic disorder that generally affects a person's lungs and digestive system. The foundation has raised over $1 million, largely from viral fundraising through universities and video campaigns. The organization also participates in exploratory research initiatives aimed at understanding and advancing towards a cure.
Jeffrey Leiden is an American physician, scientist and businessman who is the executive chairman of Vertex Pharmaceuticals, a biotechnology company based in Boston, Massachusetts. He was initially appointed to the board of directors of the company in 2009 and was CEO and president from February 2012 to March 2020.
Robert Williamson is a retired British-Australian molecular biologist who specialised in the mapping, gene identification, and diagnosis of human genetic disorders.
Jane Carolyn Davies is a British physician who is Professor of Paediatric Respirology at Imperial College School of Medicine. She is an Honorary Consultant at the Royal Brompton and Harefield NHS Foundation Trust.
Elexacaftor/tezacaftor/ivacaftor, sold under the brand names Trikafta and Kaftrio, is a fixed-dose combination medication used to treat cystic fibrosis. Elexacaftor/tezacaftor/ivacaftor is composed of a combination of ivacaftor, a chloride channel opener, and elexacaftor and tezacaftor, CFTR modulators.
Underrepresented populations, especially black and hispanic populations with cystic fibrosis are often not successfully diagnosed. This is in part due to the minimal dissemination of existing data on patients from these underrepresented groups. While white populations do appear to experience a higher frequency of cystic fibrosis, other ethnicities are also affected and not always by the same biological mechanisms. Thus, many healthcare and treatment options are less reliable or unavailable to underrepresented populations. This issue affects the level at which public health needs are being met across the world.
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