A second-generation "hypoxia in a pill" rescues neurodegenerative phenotypes across distinct mouse models

Hong Wang et al., A second-generation "hypoxia in a pill" rescues neurodegenerative phenotypes across distinct mouse models, Proceedings of the National Academy of Sciences (2026). DOI: 10.1073/pnas.2618654123

GBT601/PT2399 Halts Neurological Disease Phenotypes in Friedreich’s Ataxia Mouse Model. Here, we tested whether the GBT601/PT2399 combination could mimic the effects of inhalational hypoxia to similarly halt neurological symptoms in the shFxn mice once they began to exhibit signs of advanced debility. Hence, these studies indicate that initiating the combination treatment at 24 wk of age, can halt further progression of the ataxia phenotype, though in contrast to 11% FIO2 (13), is unable to reverse these motor phenotypes.