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Preclinical studies using transgenic models consistently report improvements in cognition and neuropathology, and early clinical trials with peptide-based compounds have provided encouraging, albeit preliminary, results

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Side effects reported include daytime grogginess, headaches, or hormonal shifts

Symptoms, which typically emerge before six months of age, include failure to thrive, decreased appetite, lethargy, anemia, and GI issues like diarrhea

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