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Wednesday – Cystic Fibrosis: Precision Treatment of a Rare Monogenic Disease Mini-Symposium

October 4, 2023 @ 3:00 pm - 5:00 pm

PPMH mini-symposium poster Oct2023

Join the UNC Program for Precision Medicine in Health Care (PPMH) for Cystic Fibrosis: Precision Treatment of a Rare Monogenic Disease, a free virtual mini-symposium from 3:00-5:00pm on Wednesday October 4, 2023. Cystic fibrosis (CF) is caused by two single-nucleotide changes to a single gene, the cystic fibrosis transmembrane conductance regulator (CFTR). The CFTR gene contains the instructions for making a protein that carries water and salt across cell membranes. When the CFTR gene is not working correctly, mucus can accumulate in the lungs and digestive system. The speakers in this mini-symposium will address the process of developing new CF therapies, methods for determining which therapy will most benefit individual patients with CF, and how the EHR can be leveraged to streamline treatment for patients with CF.



Developing small-molecule therapies to rescue rare CFTR variants

Rhianna Lee, PhD

Postdoctoral associate, Duke University, Cell Biology Department


Personalized medicine, the fight against antibiotic resistance, and precision dosing

Gauri Rao, PharmD

Associate Professor, USC Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California

Adjunct Associate Professor, UNC Eshelman School of Pharmacy, UNC Chapel Hill


From Data to Dosing: Streamlining CF modulator choices

Michael Adams, MD

Clinical Instructor, Division of Genetics & Metabolism, Department of Pediatrics, UNC School of Medicine


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Contact with any questions.


October 4, 2023
3:00 pm - 5:00 pm
Event Category:
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