inherited disease :
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- For new Rhode Island firm, peptides hold hope for disease-altering cystic fibrosis treatments
It’s been nearly two-and-a-half decades since the gene associated with the inherited disease cystic fibrosis was discovered, paving the way for disease-modifying drugs. In that time, only one such drug has been approved in the U.S. and Europe — Vertex’s Kalydeco. And that drug is only approved for the 4 percent of CF patients who [...]
June 10, 2013 - MedCity News - Carlsbad toddler is poster child for cystic fibrosis bike ride
A genetic disease that affects both the lungs and digestive system claims lives every day. Cystic fibrosis, also known as 65 Roses, is an inherited disease that affects quality of life. The CF gene mutation is passed to children by their parents. Genes also decide a lot of other body characteristics, including height, hair color and eye color.
June 9, 2013 - Carlsbad Current-Argus