脑瘫
医学
外显子组测序
神经影像学
基因检测
智力残疾
精神运动学习
儿科
全球发育迟缓
神经系统检查
神经发育障碍
运动障碍
家族史
体格检查
物理医学与康复
精神科
表型
病理
内科学
遗传学
认知
自闭症
基因
疾病
生物
作者
Marzieh Nejabat,Soroor Inaloo,Afsaneh Taghipour Sheshdeh,Shima Bahramjahan,Fatima Masoomi Sarvestani,Pegah Katibeh,Hamid Nemati,Kaoru Tabei,Mohammad Ali Faghihi
标识
DOI:10.3389/fped.2021.734946
摘要
Purpose: Cerebral palsy (CP) is a heterogeneous permanent disorder impacting movement and posture. Investigations aimed at diagnosing this disorder are expensive and time-consuming and can eventually inconclusive. This study aimed to determine the diagnostic yield of next generation sequencing in patients with atypical CP (ACP). Methods: Patient eligibility criteria included impaired motor function with onset at birth or within the first year of life, and one or more of the following conditions: severe intellectual disability, positive family history, brain imaging findings not typical for cerebral palsy, abnormal neurometabolic profile, intractable seizure, normal neuroimaging despite severe psychomotor disability, after pediatric neurologist assessment including neuroimaging and biochemical-metabolic study offered for genetic study. Results: Exome sequencing was done for 66 patients which revealed pathogenic, likely pathogenic, and variants of unknown significance in 36.2, 9, and 43.9%, respectively. We also found 10 new mutations and were able to suggest specific and personalized treatments for nine patients. We also found three different mutations with different phenotypical spectrum in one gene that have not been reported for cerebral palsy. Conclusion: An accurate history and physical examination and determination of patients with atypical cerebral palsy for doing exome sequencing result in improved genetic counseling and personalized management.
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