Cystic Fibrosis Video Perspectives

Anastassios C. Koumbourlis, MD, MPH, chief in the division of pulmonary and sleep medicine and director of the Cystic Fibrosis Center at Children's National Medical Center, and professor of pediatrics at the George Washington University School of Medicine & Health Sciences, spoke with Healio about treatment options for cystic fibrosis, as well as areas of unmet needs.

In this video, Koumbourlis discussed:

• The similarities and differences when treating cystic fibrosis in infants and toddlers compared with older children, noting that while the disease is the same, it can manifest in many different ways;
• The advantages of triple treatment therapy for cystic fibrosis, commenting that this is the best therapy to give patients;
• How azithromycin or dornase alfa with hypertonic saline work against two of the most common symptoms of cystic fibrosis and are therefore ‘fairly significant’ as routine treatment;
• The importance of gene therapy and the many ways it may improve treatment for and potentially cure cystic fibrosis treatment, emphasizing that while a lot cystic fibrosis research is “semi-speculative,” the field is “definitely at a much, much better point than we were even a of couple years ago”
• The role of the newborn screen in cystic fibrosis management; and
• Areas of unmet need in the field.