In this session, we will discuss disease-altering and potentially life-saving "precision medicine" therapeutics for pediatric respiratory diseases, including genotype-based treatments and gene therapies. We will focus on cystic fibrosis, neuromuscular diseases, sickle cell disease, and asthma as examples of what the field has accomplished and the road ahead. In addition, we will discuss important ethical and cost-effectiveness considerations that researchers, Physicians, and patients will have to face as we move forward.
CME credits and/or Certificates of Completion are not provided for this session.
1- Review the current state of research towards precision and gene-based medicine in different diseases that affect respiratory health in children and adolescents
2- Understand the evidence supporting the clinical use of precision medicine approaches in pediatric lung diseases
3- Highlight areas of uncertainty regarding the use of precise therapeutics, including eligibility, cost, and other ethical considerations
Erick Forno, MD, MPH, ATSF: Introduction
Gregory S. Sawicki, MD, MPH: Precision Medicine in Action: The Promise of Genetic Based Therapies in Cystic Fibrosis
Mymy Buu, MD: Precision Therapeutics for Spinal Muscular Atrophy and Other Neuromuscular Diseases
Anke H. Maitland-van-der-Zee, PharmD, PhD, AMC: Precision Medicine in Childhood Asthma: The Long Road Ahead
Ann Chen Wu, MD, MPH: Cost-Effectiveness and Ethics Considerations of Precision Medicine for Pediatric Lung Diseases
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