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In animal models, homozygous deletion of IGF-1 or IGF-1R genes results in high lethality and severe CNS deficits, including reduced brain size and loss of specific neurons, highlighting the essential role of IGF-1 signaling in neurodevelopment (Holzenberger et al., 2003)

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However, these are merely early signals, and robust finding regarding efficacy cannot be made until the results of larger randomized controlled trials with determining endpoints come about

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