Does Treatment with Diazoxide or a novel form of Diazoxide Choline elevate the plasma glucose levels in GLUT1 Deficiency Syndrome thus potentially increasing glucose transport the blood brain barrier?

MDBRGLUT1

Feb 1

Awardee: Paul Thornton

Institution: Cook Children's Medical Center

Grant Amount: $60,367

Funding Period: February 1, 2025 - January 31, 2026

Summary:

We propose to study currently available Diazoxide and a novel drug, Diazoxide Choline which is a more palatable tablet formulation, to see if we can elevate the plasma glucose safely and effectively in children and adults with Glut 1 Deficiency Syndrome. We aim to 1) determine if Diazoxide and Diazoxide Choline increase plasma glucose levels measured by continuous glucose monitoring; 2) compare effects of equivalent doses of Diazoxide and Diazoxide Choline on CGM measured glucose; and 3) evaluate safety and tolerability of both forms of Diazoxide.

MDBRGlut12024-1

Million Dollar Bike Ride

Does Treatment with Diazoxide or a novel form of Diazoxide Choline elevate the plasma glucose levels in GLUT1 Deficiency Syndrome thus potentially increasing glucose transport the blood brain barrier?

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