News

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

Cystic fibrosis (CF) patients, caregivers and supporters are invited to participate in Rose Up, a Sept. 17 virtual event aimed at uniting the CF community and raising awareness and funds to help find a cure. The hope is that the Cystic Fibrosis Foundation (CFF) effort, now in its…

An assessment called the lung clearance index could be used to help identify people with cystic fibrosis (CF) who are at high risk for worsening lung function, a study has found. The study reported on factors associated with a worsening lung clearance index (LCI) over time in CF patients,…

The triple-combination therapy Trikafta significantly improved lung function and life quality in a global Phase 3 trial that enrolled people with cystic fibrosis (CF) caused by one F508del mutation, and one gating mutation or one residual function mutation, in the CFTR gene. Data from the…

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…

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…