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Progress toward acetate supplementation therapy for Canavan disease: glyceryl triacetate administration increases acetate, but not N-acetylaspartate, levels in brain

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The complete approval process consists of five major stages: preclinical toxicology studies, Phase I human safety and tolerance testing, Phase II efficacy evaluation, Phase III large-scale controlled clinical trials, and GMP manufacturing site audits

A discrete Gamma distribution with Invariant (G + I) sites was used to model evolutionary rate differences among sites