The clinical and genetic profiles of spinal muscular atrophy with respiratory distress type 1 and identification of a novel mutation in IGHMBP2 in China

BackgroundSpinal muscular atrophy with respiratory distress type 1 (SMARD1, OMIM #604320) is a rare autosomal recessive hereditary degenerative motor neuron disease caused by mutations in IGHMBP2. There is a lack of data from China. This study investigated the clinical characteristics and genetic ro...

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Bibliographic Details
Main Authors: Yanjun Wang, Ya Yang, Jingjing Wang, Qian Han, Nana Zhai, Shufang Xiao
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-07-01
Series:Frontiers in Genetics
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Online Access:https://www.frontiersin.org/articles/10.3389/fgene.2025.1594265/full
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