2016 Symposium Presentations
Developing Therapeutics for the Treatment of Orphan Diseases with Significant Unmet Medical Need
Robert D. Steiner, MD, CMO
Acer Therapeutics
Gene Replacement And Genome Editing In The Treatment Of Liver Metabolic Diseases
James M. Wilson, MD, PhD.
Perelman School of Medicine, University of Pennsylvania
MSUD – A sense of Progress 1986-2016 Part I
Dr. Holmes Morton MD – Pediatrician
Central Pennsylvania Clinic, Belleville, Pennsylvania
MSUD – A sense of Progress 1986-2016 Part II
Dr. Holmes Morton MD – Pediatrician
Central Pennsylvania Clinic, Belleville, Pennsylvania
Phenylbutyrate Therapy for MSUD
Brendan Lee, MD, PhD.
Department of Molecular and Human Genetics Baylor College of Medicine
TESTIMONIALS:
MSUD Parents
Testimonials:
MSUD Parents
2016 Symposium Presentations (No Videos Available)
Metabolism, Infection and Immunity in Inborn Errors of Metabolism (PDF)
Peter J. McGuire MS, MD, National Human Genome Research Institute
Dietary Management on a Dime (PDF)
Julie McClure, MPH, RD – Metabolic Dietitian, UNC-CH
Enhancing Pediatricians Awareness of MSUD Infants Health in China (PDF)
Xinlin Hou, MD,PhD