Long-term safety: 94% of 48 TSC patients with refractory epilepsy maintained improved seizure control over 4 years; safe and tolerable. Adverse effects decreased over time. White matter modification: Everolimus pharmacologically… Click to show full abstract
Long-term safety: 94% of 48 TSC patients with refractory epilepsy maintained improved seizure control over 4 years; safe and tolerable. Adverse effects decreased over time. White matter modification: Everolimus pharmacologically modifies the genetic defect of TSC (including normal-appearing white matter) in 28 patients for 12–18 months. Longer exposure and younger age (<10 years old) are associated with greater effects. Dosing and response: 5–7 ng/mL initially and 5–15 ng/mL if inadequate clinical response. It is more difficult for patients with higher baseline seizure frequency to respond.
               
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