Study on the Treatment of Tuberous Sclerosis with Rapamycin Nanomicelles Based on Abdominal Ultrasound.

Abstract

In recent years, mTOR signaling pathway has been found to be the main bridge between TSC1/TSC2 gene mutation and tuberous sclerosis phenotype. Although mTOR inhibitors have been reported to treat tuberous sclerosis in foreign countries, there is still a lack of long-term follow-up results and clinical treatment experience in children. Therefore, research at home and abroad is actively focusing on the mTOR signaling pathway to further clarify the pathogenesis of the disease, and from a clinical point of view, to summarize the clinical data of more patients treated with mTOR inhibitors, to conduct a long-term follow-up and exploration of rapamycin treatment, and to summarize mature treatment experience. This is also the research hotspot of tuberous sclerosis. Based on the study of the treatment of tuberous sclerosis patients with rapamycin nanomicelles by abdominal ultrasound, the therapeutic effect and safety were compared and evaluated through the observation and description of the clinical seizure control and the recovery of EEG peak out of rhythm in children with tuberous sclerosis and infantile spasm.