Persistent neuromuscular junction transmission defects in adults with spinal muscular atrophy treated with nusinersen

OBJECTIVE: Spinal muscular atrophy (SMA) is a motor neuron disease caused by low levels of survival motor neuron (SMN) protein. Prior work in models and patients has demonstrated electrophysiological and morphological defects at the neuromuscular junction (NMJ). Therapeutic development has resulted...

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Detalles Bibliográficos
Autores principales: Arnold, W David, Severyn, Steven, Zhao, Songzhu, Kline, David, Linsenmayer, Matthew, Kelly, Kristina, Tellez, Marco, Bartlett, Amy, Heintzman, Sarah, Reynolds, Jerry, Sterling, Gary, Weaver, Tristan, Rajneesh, Kiran, Burghes, Arthur H M, Kolb, Stephen J, Elsheikh, Bakri
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2021
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8362737/
https://www.ncbi.nlm.nih.gov/pubmed/34466806
http://dx.doi.org/10.1136/bmjno-2021-000164

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