cystic fibrosis

A national newborn screening (NBS) program in the Republic of Ireland suggests that the incidence of cystic fibrosis and CF carriers in the country is lower than previously estimated, a study found. It also shows that the Gly551Asp mutation is almost three times more common among newborns in Ireland…

ELX-02, an investigational treatment of cystic fibrosis due to nonsense mutations, has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). This designation is given to treatments with the potential to improve care for rare diseases, defined as those affecting fewer than 200,000 people in…

The Scottish Government and Vertex Pharmaceuticals have reached a deal to make Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor) available for people with cystic fibrosis (CF) once the medication receives its European license. This follows similar agreements in the three other U.K. countries, England, Wales, and Northern…

People with cystic fibrosis (CF) are more frequently infected with oral Candida fungus and carry a higher number of fungi than healthy individuals, a study has found. All fungal species identified in the study’s CF patients were susceptible to currently available anti-fungal medications. The study, “Oral…

The government of  Northern Ireland reached a deal with Vertex Pharmaceuticals to make Kaftrio (ivacaftor/tezacaftor/elexacaftor) available for cystic fibrosis (CF) patients, once the medication receives its European license. The agreement follows similar ones that the company made recently with England and Wales.

A new study will assess the real-world effects of Vertex Pharmaceutical‘s Trikafta, a triple combo of elexacaftor, tezacaftor, and ivacaftor approved by the U.S. Food and Drug Administration (FDA) for treating up to 90% of all people with cystic fibrosis (CF). The $3.63 million study (3.06…

Researchers have developed the first thread-based flexible patch that can be sewn into clothing to monitor — in real time — several molecules in sweat, including chloride ions, which are dysregulated in cystic fibrosis (CF) patients. The study describing that finding, “Thread-based multiplexed sensor patch for…

This year’s Cystic Fibrosis Foundation (CFF) Impact Grants, which provide up to $10,000 in funding, will support and empower the cystic fibrosis (CF) community through four new virtual programs focused on engaging and connecting patients. Now in its fifth year, the program helps individuals and organizations whose projects…

After raising more than $268,828 ($360,000 Canadian dollars) since 2017 for Cystic Fibrosis Canada, thyssenkrupp Elevator Canada received the nonprofit’s 2019 National Champion Award. The award recognizes companies, service groups, and other entities that have a presence in at least three regions of Canada and have provided exceptional…

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