At the 2016 North American Cystic Fibrosis Conference (NACFC) held on Oct. 27-29 in Orlando, Fla., the Cystic Fibrosis Foundation announced the recipients of seven awards given to members of the research and medical community whose achievements have helped advance cystic fibrosis research and care.
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Theratyping matches therapies, or medications, to specific types of mutations. The primary goal of theratyping is to identify which mutations respond to certain CFTR modulators, thereby helping people with rare CFTR mutations gain access to already approved modulators quickly and safely.
Some genetic diseases, such as cystic fibrosis, are caused by mutations in a single gene. A gene contains DNA “letters” that spell out the instructions to make a specific protein. When the protein isn't made correctly, it can lead to a cascade of problems.
Carrier (or genetic) testing not only plays a key role in the diagnosis of cystic fibrosis, but testing also allows parents to find out what their chances are of having a child with CF to help inform important family planning decisions.
The Cystic Fibrosis Foundation recently hosted the CFF Research Conference: Pushing the Frontiers, bringing together nearly 150 basic scientists, clinical researchers, graduate students and pharmaceutical company representatives to share information on recent advances and critical challenges in CF drug discovery and development.
The Cystic Fibrosis Foundation has awarded more than $23 million across 11 sites in its Research Development Program (RDP), a network of research centers that brings together top-notch scientists from different disciplines to apply their expertise to the challenges of treating cystic fibrosis.
The Cystic Fibrosis Foundation today announced an unprecedented acceleration and expansion of its research, care and patient programs for the CF community. This action is possible as a result of the sale of the Foundation's royalty rights to CF treatments developed by Vertex Pharmaceuticals Inc. The Foundation received $3.3 billion from the sale to Royalty Pharma.
The Cystic Fibrosis Foundation often receives questions about the safety and effectiveness of potential CF therapies. We have recently become aware of reports that certain companies are offering stem cell treatments to people with CF -- often for large fees -- based on claims that these procedures are clinically beneficial to people with CF.
Leaders of the Cystic Fibrosis Foundation and dedicated volunteers from the CF community came together last week around a shared commitment to not stop working until a cure is found for every person living with cystic fibrosis.
The objective of this award is to support excellent cystic fibrosis-related research projects that have been approved by the National Institutes of Health (or governmental funding agencies in other countries) but cannot be supported by available funds.