APPTT
CLIN
Emily DeBoer, MD
University of Colorado / Children's Hospital Colorado
Assem Ziady, Ph.D.
Bone Marrow Transplantation and Immunodeficiency
Cincinnati Children’s Hospital Medical Center
The advent of high efficacy modulator therapies (HEMT) of CFTR has transformed CF care and also changed the biology of the disease. However, lung function can continue to decline following HEMT. The molecular basis for disease progression in persons with CF on HEMT is not understood. Clinical and molecular biomarkers following therapy remain important to predict disease progression and/or point to disease associated pathways that may be sites of intervention. Furthermore, biomarkers can aid in identifying patients that would derive maximum benefit from HEMT and those that may be good candidates for therapy simplification. For this session, we will hear presentations on the examination of clinical and molecular marker following therapy.
Speaker: Joshua D. Chandler, PhD (he/him/his) – Emory University School of Medicine
Speaker: Emily J. Skala, BS – Cincinnati Children's Hospital Medical Center
Speaker: Nicola Robinson, MBChB(Hons) MRCPUK – Centre for Inflammation Research, University of Edinburgh
Speaker: Christine Højte, MD (she/her/hers) – Copenhagen Cystic Fibrosis Center, Rigshospitalet, Copenhagen, Denmark
Speaker: Elpis Hatziagorou, PhD, MD – Aristotle University of Thessaloniki