Welcome and introduction |
Rachel Powers RD – Nutricia, Denise Kolivoski and Sandy Bulcher |
Driving Research For MSUD |
Karen Dolins, RD, EdD |
Investigating Liver and Brain Gene Therapy Strategies for MSUD |
John Counsell PhD |
Drug Discovery for Maple Syrup Urine Disease |
Max Wynn PhD |
Changing the Trajectory of MSUD Research with the CoRDS Registry |
Ben Fored, MDB and Karen Dolins, RD, EdD |
Brain hemodynamics in patients with MSUD using fNIRS |
Kosar Khaksari PhD |
Novel models to study chronic neurological sympytoms in branched chain amino acid disorders |
Rebecca Ahrens-Nicklas MD |
MSUD incidence estimates from publicly available datasets |
Rebecca Ahrens-Nicklas MD |
Medical Management of MSUD |
Nicholas Ah Mew MD |
Welcome to the 2018 MSUD Symposim in Pittsburgh, PA |
Sandy Bulcher, RN, MSUD Symposium Coordinator |
MSUD Cooking Demonstration |
Chef Neil, Vitaflo International |
Uncovering the Metabolic and Nutrient Sensing Dysfunction in MSUD |
Arvind Ramanathan, PhD, Buck Institute |
Medical Management of MSUD |
Nicholas Ah Mew MD, Children’s National Health System |
Nutrition Management of MSUD |
Rani Singh PhD, RD, Emory University |
Genetic Therapies For MSUD |
James Wilson, MD, PhD, UPenn Orphan Disease Center |
Gene Editing and Cell Transplantation for the Treatment of Liver Disease |
Steven Strom, PhD, Karolinska Institute |
Using Patient-Derived Cells to Discover New Treatments for MSUD |
Brian Wamhoff, PhD, HemoShear Therapeutics |
MSUD Teen and Adult Panel |
MSUD Teen and Adult Panel |
MSUD Professional Panel |
Dr. Ah Mew, Dr. Singh, and Dr. Bjoraker discuss life with MSUD and treatment for MSUD in a Q&A format. |
Advocacy for the MSUD Community |
Dr. Karen Reznik Dolins, EdD, RD, CSSD, CDN |
Advocacy for the MSUD Community |
Chad Farquharson, MSUD Parent |
Beyond Survival – What do we mean by Successful Treatment? |
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 |
Gene replacement and genome editing in the treatment of liver metabolic diseases |
James M. Wilson, MD, PhD Director, Orphan Disease Center Director, Gene Therapy Program Medicine and Pediatrics, Perelman School of Medicine, University of Pennsylvania |
Developing Therapeutics for the Treatment of (Ultra) Orphan Diseases |
Robert D. Steiner, MD, CMO – Acer Therapeutics Clinical Professor, University of Wisconsin |
2016 Symposium – MSUD Patients |
MSUD patients speaking about their experiences. |
2016 Symposium – MSUD Parents |
Parents of MSUD children speaking about their experiences. |