The Rare Disease Cures Accelerator-Data and Analytics Platform — dubbed RDCA–DAP — has received new datasets that promise to…
Articles by Patricia Inácio, PhD
A 3-year-old boy who carries a new combination of two mutations in the CFTR gene, but with no clinical signs…
A new small molecule, called VOMG, showed good antimicrobial activity against Mycobacterium abscessus, a bacterium that can cause serious infections…
Men and patients diagnosed with cystic fibrosis (CF) earlier in life have higher lung function and greater lung function…
Destiny Pharma is launching a research program to test a potential therapy derived from its XF drug platform…
Combining telehealth with home spirometry to assess lung function, along with some in-person visits to the doctor’s office, was as…
Trikafta (elexacaftor/tezacaftor/ivacaftor) significantly lowered several pro-inflammatory blood markers with as early as one month of use in cystic…
Vertex Pharmaceuticals and England’s National Health Service (NHS) have reached an agreement that allows all current and future eligible…
Most parents of newborns diagnosed with cystic fibrosis (CF) or a different disease as part of U.S.
The use of the approved medication Trikafta (elexacaftor/tezacaftor/ivacaftor) was shown to significantly increase weight in people with cystic fibrosis-related…