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与NFU1突变相关的新型痉挛性截瘫表型。

New spastic paraplegia phenotype associated to mutation of NFU1.

作者信息

Tonduti Davide, Dorboz Imen, Imbard Apolline, Slama Abdelhamid, Boutron Audrey, Pichard Samia, Elmaleh Monique, Vallée Louis, Benoist Jean François, Ogier Heléne, Boespflug-Tanguy Odile

机构信息

Paris Diderot University - Sorbonne Paris Cité; Inserm U1141, DHU PROTECT, Robert Debré Hospital, Paris, France.

Department of Brain and Behavioral Sciences, Unit of Child Neurology and Psychiatry, University of Pavia, Pavia, Italy.

出版信息

Orphanet J Rare Dis. 2015 Feb 8;10:13. doi: 10.1186/s13023-015-0237-6.

Abstract

Recently an early onset lethal encephalopathy has been described in relation to mutations of NFU1, one of the genes involved in iron-sulfur cluster metabolism. We report a new NFU1 mutated patient presenting with a milder phenotype characterized by a later onset, a slowly progressive spastic paraparesis with relapsing-remitting episodes, mild cognitive impairment and a long survival. The early white matter abnormalities observed on MRI was combined with a mixed sensory-motor neuropathy in the third decade. Our case clearly suggests the importance of considering NFU1 mutation in slowly evolving leukoencephalopathy with high glycine concentration.

摘要

最近,已经描述了一种与铁硫簇代谢相关基因之一的NFU1突变有关的早发性致死性脑病。我们报告了一名新的NFU1突变患者,其表现出较轻的表型,特征为发病较晚、缓慢进展的痉挛性截瘫伴复发-缓解发作、轻度认知障碍和长期存活。在第三个十年中,MRI上观察到的早期白质异常与混合性感觉运动性神经病变并存。我们的病例清楚地表明,在高甘氨酸浓度的缓慢进展性白质脑病中考虑NFU1突变的重要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/57db/4333890/2df22ff81c79/13023_2015_237_Fig1_HTML.jpg

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