Abstract
Pioglitazone as a novel therapeutic approach in chronic granulomatous disease
Highlights
To the Editor: Chronic granulomatous disease (CGD) is a rare genetic disease caused by defects in genes encoding the subunits of the nicotinamide adenine dinucleotide phosphate oxidase complex.[1]
Pioglitazone, a peroxisome proliferator–activated receptor gamma agonist approved for type 2 diabetes, was reported in this journal to induce mitochondrial reactive oxygen species (ROS) production in mice with X-chromosome–linked CGD (X-CGD)
For the first time, the use of pioglitazone as a novel therapeutic approach in a 5-month-old boy with X-CGD experiencing multiple severe infections
Summary
To the Editor: Chronic granulomatous disease (CGD) is a rare genetic disease caused by defects in genes encoding the subunits of the nicotinamide adenine dinucleotide phosphate oxidase complex.[1]. Because of the underlying general conditions of the patient with persistent pulmonary distress requiring protracted noninvasive ventilation, severe delay in neuromuscular development, and failure to thrive, HSCT was postponed despite availability of an HLA-identical sibling.
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