AF7- Administration of Elexacaftor/Tezacaftor/Ivacaftor in a CFTR Carrier Mother for Treatment of Meconium Ileus in a F508del Fetus with Extension of Therapy into the Postpartum Period
Speaker: Beth A. Smith, MD – Jacobs School of Medicine and Biomedical Sciences, University at Buffalo
Speaker: Stephanie S. Filigno, PhD – Cincinnati Children's Hospital Medical Center, Divisions of Behavioral Medicine and Clinical Psychology & Pulmonary Medicine
W19.1- Impact of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on healthcare resource utilization (HCRU) over three years in LONGITUDE – a UK CF Registry observational study
W20.2- A Phase II study (CLAIRAFLY®) evaluating safety, tolerability, pharmacokinetics and pharmacodynamics of the DPP1 (CatC) inhibitor BI 1291583 in cystic fibrosis: Effects of CFTR modulator therapy use
Location: Flex A (Level 2)
Speaker: Marcus A. A. Mall, MD – Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine
10:15 AM - 12:15 PM PDT
W20.3- Safety, tolerability, and pharmacokinetics of novel NBD1 stabilizers SION-719 and SION-451 from two phase 1 first-in-human studies
W20.4- Nutritional decline and glycemic variability as early clinical indicators in pediatric CF patients undergoing CGM: a retrospective cohort analysis
W24.4- Stress less, live more: Coping and Learning to Manage Stress (CALM) telehealth sessions help alleviate severe depression and anxiety symptoms in adults with CF
W25.2- Financial & administrative impacts on people with CF, pharmacists, and pharmacy technicians at CFF-accredited care centers following changes in pharmaceutical limited distribution network
W25.4- Physiologically based pharmacokinetic of elexacaftor/tezacaftor/ivacaftor in children with CF: extrapolation of the dosing regimen in children under 2 years of age
Location: Signature Room (Level 5)
Speaker: Frantz FOISSAC, PhD – UMR-S 1341, Pharmacologie Et Évaluation Des Thérapeutiques Chez L'enfant Et La Femme Enceinte Université Paris Cité, Paris, France.
10:15 AM - 12:15 PM PDT
W25.5- Modeling a single variable-time measurement of elexacaftor/tezacaftor/ivacaftor in adults
Location: Signature Room (Level 5)
Speaker: Silvia Caceres, MS – University of North Carolina- Chapel Hill/ National Jewish Health
W26.4- Pregnancy and early parenthood does not have a detrimental impact on CF-health status in women under a dedicated CF Maternal Health Service in the elexacaftor/tezacaftor/ivacaftor era
W28.1- An evaluation of fecal metabolomics and markers of intestinal integrity in individuals with advanced CF liver disease (aCFLD) and the effect of elexacaftor-tezacaftor-ivacaftor (ETI)
Location: Ballroom 2 (Level 5)
Speaker: Nicole Green, M.D. – Division of Gastroenterology and Hepatology, Department of Pediatrics, University of Washington and Seattle Children's Hospital, Seattle, WA, USA
10:15 AM - 12:15 PM PDT
W28.2- Multivariate classification of gut symptoms in people with CF identifies four phenotypes
W29.3- Targeted eRF3a degradation amplifies nonsense mutation readthrough in a CF murine model
Location: Terrace Suite (Level 4)
Speaker: Lisa Nicholson – Case Western Reserve University.
10:15 AM - 12:15 PM PDT
W29.4- Small molecules as Aminoglycoside Enhancers to promote readthrough for the treatment of CF
Location: Terrace Suite (Level 4)
Speaker: Corinne E. Augelli-Szafran, PhD, MS – Heersink School of Medicine, University of Alabama at Birmingham, Department of Biochemistry and Molecular Genetics
10:15 AM - 12:15 PM PDT
W29.5- Discovery of non-aminoglycoside small molecules that enhance eRF3 degrader-induced translational readthrough of CFTR nonsense mutations
TPS05.4- Changes in sweat chloride concentrations following CFTR modulator treatment and association with antibiotic usage in adolescents and adults with CF
Location: Theater A Exhibit Hall
Speaker: Sivagurunathan Sutharsan, MD – Department of Pulmonary Medicine, Division of Cystic Fibrosis, University Medicine Essen -Ruhrlandklinik, University of Duisburg-Essen, Essen, Germany.
2:30 PM - 4:00 PM PDT
TPS05.5- Maximizing benefits with early CFTR modulator treatment: Lower sweat chloride is associated with improved clinical outcomes in children aged 6 to 11 years
S18.4- Weight Inclusive Approach to Counseling Patients and Caregivers
Location: Flex A (Level 2)
Speaker: Stephanie S. Filigno, PhD – Cincinnati Children's Hospital Medical Center, Divisions of Behavioral Medicine and Clinical Psychology & Pulmonary Medicine
2:30 PM - 4:30 PM PDT
S19--Don’t Sweat It: Navigating the Relationship Between Sweat Chloride & Clinical Outcomes in CF | 435
S19.3- Sweat Signals: Use of Sweat Chloride in Clinical Trials of CFTR Modulators
Location: 435
Speaker: Rachel Linnemann, MD – Department of Pediatrics, Division of Pulmonology, Asthma, Cystic Fibrosis, and Sleep, Emory University School of Medicine
2:30 PM - 4:30 PM PDT
S19.4- Promise and Pitfalls in Sweat Chloride Use for Clinical Monitoring and Therapeutics Development
Speaker: Nicole Green, M.D. – Division of Gastroenterology and Hepatology, Department of Pediatrics, University of Washington and Seattle Children's Hospital, Seattle, WA, USA
2:30 PM - 4:30 PM PDT
S20.3- Beyond the Channel: Immune Implications of CFTR Modulation