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Spinal muscular atrophy with respiratory distress type 1: Clinical phenotypes, molecular pathogenesis and therapeutic insights

Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is a rare autosomal recessive neuromuscular disorder caused by mutations in the IGHMBP2 gene, which encodes immunoglobulin μ‐binding protein 2, leading to progressive spinal motor neuron degeneration. We review the data available in t...

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Dades bibliogràfiques
Publicat a:J Cell Mol Med
Autors principals: Saladini, Matteo, Nizzardo, Monica, Govoni, Alessandra, Taiana, Michela, Bresolin, Nereo, Comi, Giacomo P., Corti, Stefania
Format: Artigo
Idioma:Inglês
Publicat: John Wiley and Sons Inc. 2019
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6991628/
https://ncbi.nlm.nih.gov/pubmed/31802621
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/jcmm.14874
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