Although 90-95 percent of people with cystic fibrosis are expected to benefit from CF transmembrane regulator (CFTR) modulators, 5 percent of the population will still need alternative therapies to address the underlying cause of their disease. Learn more about the research that the Cystic Fibrosis Foundation is doing to find treatments for the 5 percent of people with these rare and nonsense mutations.
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Each year, Great Strides Ambassadors inspire walk participants across the country and are the face of the adult cystic fibrosis community. Meet the 2017 Ambassadors, Lea Faraone and Willem Wery, and learn why they stride for CF.
This year, theratyping, the process of matching medications with mutations, enabled more people with cystic fibrosis who have rare mutations to benefit from modulators. In the first plenary at the NACFC, we heard more about how theratyping will be used, as well as other future advances coming in CF treatment.
As both a researcher and a person with cystic fibrosis, it is an amazing experience to watch cells with rare CF mutations respond to drugs in the lab. Knowing the scientific basis for my treatments not only gives me a sense of control, but it encourages me to do my treatments.
Prime Medicine’s prime editing technology has the potential to bring genetic therapies to all people with cystic fibrosis, ultimately paving the way to a cure.
The additional funding will support a Phase 2 clinical trial for an inhaled antisense oligonucleotide drug for people with cystic fibrosis who have the splicing mutation 3849+10Kb C-to-T.