News

Kalydeco Approved in Canada to Treat Infants 4 Months and Older

Health Canada has extended the use of Kalydeco (ivacaftor) to treat babies, starting at 4 months old, who have cystic fibrosis (CF) caused by certain mutations. The specific gating mutations covered are: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. Kalydeco, Vertex Pharmaceuticals, was previously approved in…

65 Roses Ladies Golf Classic Benefits CF Canada

The Calgary and Southern Alberta Chapter of Cystic Fibrosis Canada has announced that this year’s 65 Roses Ladies Golf Classic, a women-only golf tournament to raise funds and awareness for cystic fibrosis (CF), will take place Sept. 13 at Earl Grey Golf Club in Calgary. The event offers…

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…