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Keynote By Chief of Pediatrics

Dr. Wendy Chung
Chief of Pediatrics
Wendy Chung, MD, PhD is the Chief of Pediatrics at Boston Children’s, Mary Ellen Avery Professor at Harvard Medical School, and President of Children’s Hospital Pediatric Associates. She is a renowned clinical and molecular geneticist with over 20 years of experience leading NIH-funded research on a vast array of diseases and over 50 monogenic disorders — 3 of which bear her name. Dr. Chung led the adoption of nationwide newborn screening for spinal muscular atrophy and led GUARDIAN, a study on newborn whole-genome sequencing. She has authored over 600 peer-reviewed publications and earned numerous accolades, including the Rare Impact Award.
Antisense Oligonucleotide Therapy for
KIF1A-Associated Neurological Disorder
In this keynote, Dr. Chung will describe the patient’s journey and role of genomics in KIF1A-Associated Neurological Disorder. Dr. Chung will also review the development of a novel antisense oligonucleotide therapy and discuss how this story could be replicated at scale for more patients.
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