Researchers Explore Trikafta in Younger Patients

Researchers at the Children’s Hospital Colorado Breathing Institute are participating in four new studies to evaluate the long-term impact of Trikafta (elexacaftor, tezacaftor, and ivacaftor) in people with cystic fibrosis (CF) ages 6 and older, its effectiveness in infants and children younger than 6, and to find alternative…

Adrulipase Alfa Likely Global Name for CF Non-porcine PERT Candidate

The World Health Organization (WHO) proposed a compound name to better identify MS1819, an investigational therapy for exocrine pancreatic insufficiency in people with cystic fibrosis (CF), with scientists globally. MS1819 may soon be called “adrulipase alfa.” The WHO is expected to make a final decision on the candidate’s International Nonproprietary…

CF Canada Urges Adoption of New Care Guidelines, Access to Trikafta

Cystic Fibrosis Canada (CFC) is calling on provinces and territories across the country to adopt the latest standardized care guidelines and speed patient access to Trikafta, a triple-combination and “highly impactful” disease-modifying therapy for almost all with cystic fibrosis (CF). The guidelines, “Initiation, Monitoring and…

MS1819 Plus PERT Improved Fat Absorption in Severe EPI

Combining investigational therapy MS1819 with standard pancreatic enzyme replacement therapy (PERT) can improve fat absorption in people with cystic fibrosis (CF) who have severe exocrine pancreatic insufficiency (EPI), top-line results from a Phase 2 clinical trial show. “A safe and effective therapy that allows CF patients to gain control over…

Register Now for Global Genes’ RARE Patient Advocacy Summit

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

Lung Transplant Biorepository, Registry Targets Patient Outcomes

The Cystic Fibrosis Foundation (CFF) and the Cleveland Clinic are launching the Cystic Fibrosis Lung Transplant Consortium (CFLTC) Biorepository and Registry, aimed at supporting research into understanding the factors that influence lung transplant outcomes. “The biorepository and registry for lung transplant recipients represents an exciting leap forward in…

Featured Column

Little Books That Communicate Big Ideas About Health

A banner for Kate Delany's "Breathe and Believe" column that depicts a pair of lungs with flowers blossoming from them.
Columnist Kate Delany shares the children’s books about health she loved as a child, as well as others she’s shared with her own children.

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