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.
An Ironman Triathlon is one of a series of long-distance triathlon races organized by the World Triathlon Corporation (WTC), consisting of a 2.4-mile (3.9 km) swim, a 112-mile (180.2 km) bicycle ride and a marathon 26.22-mile (42.2 km) run completed in that order, a total of 140.6 miles (226.3 km). It is widely considered one of the most difficult one-day sporting events in the world.
Cystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and anion channel in vertebrates that is encoded by the CFTR gene.
The Cystic Fibrosis Trust 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.
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.
The Hauptman-Woodward Medical Research Institute (HWI) is an independent, not-for-profit, biomedical research facility located in the Buffalo Niagara Medical Campus affiliated with the University at Buffalo. Its focus is on structural biology with a strong history in methods development and the application of X-ray crystallography in fundamental studies.
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.
Jason P. Lester is an endurance athlete, the author of the book Running on Faith, and the 2009 ESPY Award winner for Best Male Athlete with a Disability. He is the first disabled athlete to complete the Ultraman World Championships. He is the Founder of EPIC5 Challenge, EP1CMAN and The NEVER STOP Foundation est. 2007.
The Redstone Acceleration & Innovation Network (TRAIN) is an organization launched by FasterCures in 2004 established to create opportunities for medical research organizations to discuss and overcome research challenges that cut across all diseases. TRAIN brings together nonprofit disease research organizations to share information, as well as promote collaboration and innovation in disease research. Headed by FasterCures, TRAIN asserts that collaboration is crucial for efficient biomedical advancement. Participating researchers discuss successes, failures, and best practices that provide lessons learned and valuable ideas that could be scaled up to amplify productivity.
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.
The Canadian Lung Association is a national organization and volunteer-based health charity that supports lung health research, education, prevention of disease/disorders and advocacy in Canada.
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.
Dr. John Engelhardt is the director at the University of Iowa Center for Gene Therapy of Cystic Fibrosis, as well as the head of the department of anatomy and cell biology. He is a well known scientist and inventor who created the first cloned ferret and has made huge strides in finding the cure for cystic fibrosis.
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.
Mallory Beatrice Smith was an author and cystic fibrosis advocate.
Johanna Rommens is a Canadian geneticist who was on the research team which identified and cloned the CFTR gene, which when mutated, is responsible for causing cystic fibrosis (CF). She later discovered the gene responsible for Shwachman-Diamond syndrome, a rare genetic disorder that causes pancreatic and hematologic problems. She is a Senior Scientist Emeritus at SickKids Research Institute and a professor in the Department of Molecular Genetics at the University of Toronto.
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.
