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…
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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 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…
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
Voting is open for the 2021 Thriving Undergraduate and Thriving Graduate Scholarship, a program run by AbbVie that awards scholarships totaling $25,000 to one undergraduate and one graduate student with cystic fibrosis (CF). AbbVie selected 40 undergraduate and graduate students living with CF earlier this year, with each…
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…
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…
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…
Nathan Perdue, the father of a 2-year-old boy with cystic fibrosis (CF), in collaboration with the Kentucky Derby Museum, is raffling off his limited-edition collection of bourbons to support the Cystic Fibrosis Foundation (CFF) and its efforts to cure this disease within his son’s lifetime. The collection includes…
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…
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