2019 Million Dollar Bike Ride Awardees

Our successful fundraising drive resulted in the selection of two projects to advance MSUD research:

“A novel approach for identification of therapeutic leads to the Maple Syrup Urine Disease”

Prof. Ehud Gazit, Tel Aviv University, Israel

Using a yeast model developed for MSUD in their lab, the aim of this study is to understand basic MSUD pathology with the goal of developing novel treatments.

“Can we improve neurologic and psychiatric outcomes in MSUD? Studies in a new mouse model”

Dr. Rebecca Ahrens-Nicklas, The Children’s Hospital of Philadelphia

MSUD typically affects thought processes, mood, and behavior. This project will use a mouse model with the aim of better understanding the mechanisms by which this occurs. This knowledge is a necessary step in the development of therapies to protect the brain.

MSUD Family Support Group Supported Research

MSUD Cow Model

Researchers at the University of Massachusetts are working with the Clinic for Special Children to study gene therapy in a cow model of MSUD. The cow model is felt to have advantages over the mouse model used in most research.

Metformin and Rapamycin, Buck Institute

Researchers continue to study the effect of these substances on MSUD in hopes of developing therapies.


The MSUD Family Support Group is currently funding several research projects and we are proactively looking for researchers interested in developing new treatments or finding a cure for MSUD. Significant funding is necessary if we are to accomplish this goal.
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