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生物素酶缺乏症:患病率、影响及管理策略

Biotinidase Deficiency: Prevalence, Impact And Management Strategies.

作者信息

Canda Ebru, Kalkan Uçar Sema, Çoker Mahmut

机构信息

Department of Pediatrics, Faculty of Medicine, Ege University, Izmir, Turkey.

出版信息

Pediatric Health Med Ther. 2020 May 4;11:127-133. doi: 10.2147/PHMT.S198656. eCollection 2020.

Abstract

Biotinidase deficiency is an autosomal recessive inherited neurocutaneous disorder. Clinically untreated patients with BD can present with variable neurological and dermatological signs, such as seizures, hypotonia, feeding problems, developmental delay, hearing loss, optic atrophy ataxia, alopecia, and skin rash. Clinical findings of patients with partial BD reported in the literature show that it can occur from infancy to adulthood. Outcomes of newborn screening programs support the fact that biotin treatment started after birth prevents patients with biotinidase deficiency from developing symptoms. Presence of late-onset cases with different clinical findings indicates that there is still much to learn about BD.

摘要

生物素酶缺乏症是一种常染色体隐性遗传的神经皮肤疾病。临床上未经治疗的生物素酶缺乏症患者可能会出现多种神经和皮肤症状,如癫痫发作、肌张力减退、喂养问题、发育迟缓、听力丧失、视神经萎缩、共济失调、脱发和皮疹。文献中报道的部分生物素酶缺乏症患者的临床发现表明,该病可发生于婴儿期至成年期。新生儿筛查项目的结果证实,出生后开始进行生物素治疗可预防生物素酶缺乏症患者出现症状。存在具有不同临床症状的迟发性病例表明,关于生物素酶缺乏症仍有许多有待了解之处。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1d20/7211084/b5bbfc6ed1c3/PHMT-11-127-g0001.jpg

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