Barriers to successful genetic and pharmacologic interventions in ataxia-telangiectasia include the need for personalized treatment approaches based on patient-specific ATM mutations and phenotypes, lack of an animal model for the neurologic phenotype, and extreme rarity of disease making large-scale randomized trials difficult to perform. Ongoing efforts are needed to diagnose patients earlier, discover more effective therapies, and include more individuals in clinical trials, with the goal to lessen disease burden and to find a cure for patients with A-T....Pharmacologic treatments that target oxidative stress, inflammation, and mitochondrial exhaustion may provide neurologic benefit in A-T.