Children's Hospital Colorado

New FDA-Approved Cystic Fibrosis Medication (S3:E45)

These days, medical trainees rarely see kids with cystic fibrosis (CF) present initially to the emergency department because of advances in the diagnosis of cystic fibrosis. These exciting developments in the diagnosis and treatment of cystic fibrosis are the result of advocacy, public policy, science and evidence-based practice. Most recently, exciting new advances in therapeutics are more than just supportive care – they get to the root pathophysiology of the problem in patients with cystic fibrosis.

Listen to a pediatric pulmonologist discuss new cystic fibrosis medication

In this episode, we discuss caring for patients with cystic fibrosis and the clinical research that has led to advances in access to these breakthrough therapies with Scott Sagel, MD.

Dr. Sagel is a pediatric pulmonologist at Children’s Hospital Colorado and a Professor of Pediatrics at the University of Colorado School of Medicine.

In this episode, our experts discuss:

  • Milestones in cystic fibrosis care over the last 25 years and how they’ve translated into improved care for these patients
  • Universal adoption of newborn screening for cystic fibrosis
  • The CFTR protein and what is happening at a molecular level that eventually leads to CF
  • Development of the first CFTR modulator
  • The differences between CFTR modulators and why we’re grouping them together
  • Outcomes of patients who are being treated with CFTR modulators
  • Initiating therapy in pediatric patients before they have significant lung disease and how it protects them during the pediatric years
  • How the Cystic Fibrosis Research and Care Center gets a high level of participation in clinical studies
  • Goals for the next 10 years of CF research

Treatment of cystic fibrosis at Children’s Colorado

Our pediatric pulmonology experts at Children's Colorado are nationally recognized for their work with children who have common and complex breathing problems. Our pediatric pulmonologists, along with other Colorado physicians, pioneered many of the standard practices used to treat and diagnose pediatric respiratory disease today.

Refer a patient to Children’s Colorado.