News

#NACFC2021 – Newborn Screening Improves Real-world Outcomes

For children with cystic fibrosis (CF), newborn screening is associated with markedly improved real-world outcomes, including earlier diagnosis, greater weight and height gains, and fewer bacterial infections, new research demonstrates. In fact, infants are now diagnosed with the genetic disease at a median of 0 months…

CFF Investing Up to $110M, Partnering to Advance Gene Therapy

The Cystic Fibrosis Foundation (CFF) is partnering with Pioneering Medicines, an initiative of Flagship Pioneering, to support the development of various types of gene therapy for cystic fibrosis (CF). The foundation is ready to invest up to $110 million to move therapy candidates into clinical testing, starting with an…

#NACFC2021 – Orkambi, Symdeko May Improve Children’s Liver Health

Treatment with Orkambi (lumacaftor/ivacaftor) and/or Symdeko (tezacaftor/ivacaftor) may lessen liver damage in children and adolescents with cystic fibrosis (CF) who have liver involvement, new data indicate. “We detected improvements in validated noninvasive biomarkers of liver fibrosis [scarring] and cirrhosis in children and adolescents treated with” these therapies, the…