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Its long-term safety profile, optimal dosing, and broader clinical utility remain under investigation in ongoing Phase 3 trials

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HDAC inhibition increases acetylation of the NR3C1 gene (encoding the glucocorticoid receptor), enhancing glucocorticoid receptor sensitivity and restoring the negative feedback of the hypothalamic-pituitary-adrenal (HPA) axis

These therapies focus on helping children learn essential life skills and managing behaviors, which can have a profound impact on their quality of life