Cystic Fibrosis: Precision Treatment of a Rare Monogenic Disease Mini-Symposium
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.
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.
Speakers will address research into 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.
Presentations
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
For more information, visit www.med.unc.edu/ppmh.
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