Award
NIH Reporter #1R56HD120276-01A1
Therapeutic approaches for glutaric aciduria type I
Recipient
Duke University
Award Amount
$593,848.00
Ceiling
$593,848.00
Awarded
August 24, 2026
Identifier
1R56HD120276-01A1
This award funds research at Duke University to develop a novel therapy for glutaric aciduria type I by restoring lysine metabolism in the liver, aiming to improve patient outcomes beyond current dietary treatments.
Description
The project aims to develop targeted therapy for GA-1 by restoring lysine metabolism in the liver. The project builds on previous successful murine model studies. The overall objective is to understand better the pathophysiology of GA-1 and develop an effective treatment to improve long-term outcomes. The project duration is from September 2026 to August 2028.