2016 MSUD Symposium - Presentations PDF Print E-mail


MSUD Parents

MSUD Patients


Developing Therapeutics for the Treatment of Orphan Diseases with Significant Unmet Medical Need (PDF)
Robert D. Steiner, MD, CMO -Acer Therapeutics

Gene replacement and genome editing in the treatment of liver metabolic diseases (PDF)
James M. Wilson, MD, PhD - Perelman School of Medicine, University of Pennsylvania

MSUD - A sense of Progress 1986-2016 Part I (PDF)
Dr. Holmes Morton MD - Pediatrician, Central Pennsylvania Clinic, Belleville, Pennsylvania

MSUD - A sense of Progress 1986-2016 Part II (PDF)
Dr. Holmes Morton MD - Pediatrician, Central Pennsylvania Clinic, Belleville, Pennsylvania

Phenylbutyrate Therapy for MSUD (PDF)
Brendan Lee, MD, PhD - Department of Molecular and Human Genetics Baylor College of Medicine

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


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