Experiences from treating seven adult 5q spinal muscular atrophy patients with Nusinersen

BACKGROUND: The antisense oligonucleotide Nusinersen recently became the first approved drug against spinal muscular atrophy (SMA). It was approved for all ages, albeit the clinical trials were conducted exclusively on children. Hence, clinical data on adults being treated with Nusinersen is scarce....

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Detalles Bibliográficos
Autores principales: Jochmann, Elisabeth, Steinbach, Robert, Jochmann, Thomas, Chung, Ha-Yeun, Rödiger, Annekathrin, Neumann, Rotraud, Mayer, Thomas E., Kirchhof, Klaus, Loudovici-Krug, Dana, Smolenski, Ulrich C., Witte, Otto W., Grosskreutz, Julian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2020
Materias:
Case Series
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7059230/
https://www.ncbi.nlm.nih.gov/pubmed/32180828
http://dx.doi.org/10.1177/1756286420907803

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