NACFC 2021

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…

People with cystic fibrosis (CF) who start on the triple therapy Trikafta may be at greater risk of problems associated with excessive weight or obesity, such as metabolic syndrome — a group of disorders known to raise a risk of heart, liver, and kidney disease. “With the approval…

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…

Treatment with Trikafta can help people with cystic fibrosis (CF) to put on weight, according to new data from a small clinical trial. These findings indicate that traditional guidelines for CF nutrition — which urge a…

Treatment with SP-101, an investigational gene therapy that Spirovant Sciences is developing for people with cystic fibrosis (CF) who are unable to benefit from current treatments, effectively increased CFTR gene activity in a ferret model of the disease, the company reported. Katherine Excoffon, PhD,…

Since the approval of Trikafta in the U.S., Nationwide Children’s Hospital in Ohio has seen a steep increase in the number of women with cystic fibrosis (CF) choosing to become pregnant. “Due to the large pregnancy volume we…