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The 2.4 mg dose, which became the standard dose carried forward into phase 3 trials, produced approximately 8% to 9% weight loss in 26 weeks

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Warning signs of sleep problems in Rett syndrome can include: bedtime struggles: Having problems settling down or resisting the transition to sleep at night daytime fatigue: Needing more naps than usual or appearing excessively sleepy throughout the day nighttime disruptions: Regularly waking up at night and having difficulty falling back to sleep irregular schedules: Sleeping significantly more or less than expected or following an unpredictable sleep-wake cycle noisy breathing: Snoring or gasping during the night, which may indicate airway issues Caregivers can track these changes using a screening tool called BEARS, which evaluates sleep habits based on the childs age

Collectively, these results demonstrate that the SP1/SEPHS2 nomogram represents a reliable and clinically useful tool for predicting age-related risk, with strong discriminatory performance, good calibration, and meaningful net clinical benefit, further validating the critical role of these two hub genes in our predictive framework