Child tuberous sclerosis treatment drug

A tuberous sclerosis, children's technique used in the field of medicine to solve problems such as no treatment for the cause

Inactive Publication Date: 2015-04-08
邹丽萍 +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

However, at present, skin lesions of tuberous sclerosis, renal cysts, angiomyolipoma, and neurological complications (such as intractable epilepsy, cortical dysplasia, su

Method used

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  • Child tuberous sclerosis treatment drug
  • Child tuberous sclerosis treatment drug
  • Child tuberous sclerosis treatment drug

Examples

Experimental program
Comparison scheme
Effect test

Embodiment

[0014] Example: Clinical research on the use of rapamycin in the treatment of tuberous sclerosis in children

[0015] This clinical study is an open, prospective, self-controlled study.

[0016] The specific research methods and observation results are as follows:

[0017] (1) Case selection

[0018] Children with TSC combined with intractable epilepsy who had complete data were selected in the outpatient and ward of the PLA General Hospital. The inclusion criteria were: under 14 years old, male or female; the diagnostic criteria for TSC were based on the nodules revised by the Diagnostic Criteria Committee of the American National TSC Association in 1998. Diagnostic criteria for sexual sclerosis; children with epileptic seizures ≥ 4 times per month before enrollment, who are ineffective with antiepileptic drugs; have read the subject instructions, guardians agree and sign written informed consent; at least 24 weeks of treatment have been completed and follow-up.

[0019] E...

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PUM

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Abstract

The present invention relates to the field of medicines, and in particular discloses a child tuberous sclerosis treatment drug including rapamycin, the child tuberous sclerosis includes child tuberous sclerosis combined intractable epilepsy, child tuberous sclerosis combined cardiac rhabdomyoma, child tuberous sclerosis combined depigmentation, facial steatadenoma, and child tuberous sclerosis combined renal angioleiomyolipoma, and the drug use method is as follows: the initial dose of the rapamycin is 1mg / (m2.d), and the blood drug concentration is maintained at 5-10ng / mL.

Description

technical field [0001] The invention relates to the field of medicine, in particular to a medicine for treating tuberous sclerosis in children. Background technique [0002] Tuberous sclerosis complex (TSC) is an autosomal dominant genetic disease with an incidence of about 1:6000 and a male-to-female ratio of about 1.44:1. It is more common in children and the causative genes are TSC1 and TSC2, a number of large sample studies at home and abroad have shown that in non-selective TSC enrolled cases, the detection rate of pathogenic mutations is 69%-89%. Clinically, children often present with multiple organ involvement (such as skin, brain, eyes, kidney, liver, and heart, etc.), with facial angiofibromas, seizures, and mental decline as the main clinical features. [0003] Neurological complications of tuberous sclerosis are the most common cause of death and disability in children, 44% of children have psychomotor developmental impairment, and 30% of cases have IQ less than...

Claims

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Application Information

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IPC IPC(8): A61K31/436A61P25/08A61P35/00A61P17/00A61P25/28
CPCA61K31/436
Inventor 邹丽萍刘玉洁庞领玉石秀玉胡琳燕杨光邓艳春
Owner 邹丽萍
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