8:30 – 9:15 AM
9:15 – 9:30 AM
9:30 – 10:00 AM
10:00 – 10:30 AM
10:30 – 11:00 AM
11:00 – 1:00 PM
1:00 – 1:30 PM
1:30 – 2:00 PM
2:00 – 2:30 PM
2:30 – 3:00 PM
3:00 – 3:45 PM
3:45 – 4:00 PM
4:00 – 4:15 PM
4:15 – 4:30 PM
4:30 – 5:00 PM
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Registration and Breakfast
Welcome and Introduction
H. Clifford Lane, M.D., NIAID
Lessons and Benefits from Collaboration between the NIH and a Free-Standing Children’s Hospital
Marshall Summar, M.D., Children’s National Rare Disease Institute
The Hereditary Disorders of PropionylCoA and Cobalamin Metabolism – Past, Present, and Future
Charles Venditti, M.D., Ph.D., National Human Genome Research Institute Collaboration
The Road(s) to Genetic Precision Therapeutics in Pediatric Neuromuscular Disease: Opportunities and Challenges
Carsten Bonnemann, M.D., National Institute of Neurological Disorders and Stroke
Poster Session and Lunch
Genomic Diagnostics in Immunologic Diseases
Helen Su, M.D., Ph.D., National Institute of Allergy and Infectious Diseases
Update on Outcomes of Gene Therapy Clinical Trials for X-SCID and X-CGD, and Plans for Future Trials
Harry Malech, M.D., National Institute of Allergy and Infectious Diseases
Virus-specific T cell therapies: Broadening applicability for PID patients
Catherine Bollard, M.D., Children’s National
Using Genetic Testing to Guide Therapeutic Decisions in Primary Immune Deficiency Disease
Vanessa Bundy, M.D., Ph.D., Children’s National
Panel Discussion – Moderator Lisa M. Guay-Woodford, M.D.
Drs. Su, Malech, Bollard, and Bundy
Morgan Similuk, S.C.M., NIAID
Maren Chamorro, Parent Advocate
Underlying Mechanisms of Pediatric Food allergy: Focus on B cells
Adora Lin, M.D., Ph.D., Children’s National
Pediatric Lyme Outcomes Study – Interim Update
Roberta DeBiasi, M.D., Children’s National
Molecular Drivers and Opportunities in Neuroimmune Conditions of Pediatric Onset
Elizabeth Wells, M.D., Children’s National
Closing Reception
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