GBT1118 voxelotor analog improves skeletal muscle function, phenotype and significantly reduced serum myopathy biomarker GDF15 in sickle cell disease mice.

Abstract excerpt

We examined the effect of GBT1118, a sickle hemoglobin polymerization inhibitor on skeletal muscle grip strength and phenotype in Townes sickle cell disease (SCD) mouse model. Control male and female mice were fed with control Vehicle-chow for 2-months, while SCD male and female mice were fed with Vehicle-chow or 2-hydroxy-6-[(2S)-1-(pyridine-3-carbonyl)piperidin-2yl]methoxy (GBT1118)-chow for 2-months. We first...

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• Anemia, Sickle Cell
• Biomarkers
• Disease Models, Animal
• Hand Strength
• Muscle, Skeletal