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Nusinersen Modulates Proteomics Profiles of Cerebrospinal Fluid in Spinal Muscular Atrophy Type 1 Patients

Spinal muscular atrophy (SMA) type 1 is a severe infantile autosomal-recessive neuromuscular disorder caused by a survival motor neuron 1 gene (SMN1) mutation and characterized by progressive muscle weakness. Without supportive care, SMA type 1 is rapidly fatal. The antisense oligonucleotide nusiner...

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Detalles Bibliográficos
Autores principales:	Bianchi, Laura, Sframeli, Maria, Vantaggiato, Lorenza, Vita, Gian Luca, Ciranni, Annamaria, Polito, Francesca, Oteri, Rosaria, Gitto, Eloisa, Di Giuseppe, Fabrizio, Angelucci, Stefania, Versaci, Antonio, Messina, Sonia, Vita, Giuseppe, Bini, Luca, Aguennouz, M’hammed
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8122268/ https://www.ncbi.nlm.nih.gov/pubmed/33919289 http://dx.doi.org/10.3390/ijms22094329

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