The Need for SMN-Independent Treatments of Spinal Muscular Atrophy (SMA) to Complement SMN-Enhancing Drugs

Spinal Muscular Atrophy (SMA) is monogenic motoneuron disease caused by low levels of the Survival of Motoneuron protein (SMN). Recently, two different drugs were approved for the treatment of the disease. The antisense oligonucleotide Nusinersen/Spinraza® and the gene replacement therapy Onasemnoge...

Descripción completa

Detalles Bibliográficos
Autores principales: Hensel, Niko, Kubinski, Sabrina, Claus, Peter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Neurology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7009174/
https://www.ncbi.nlm.nih.gov/pubmed/32117013
http://dx.doi.org/10.3389/fneur.2020.00045

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7009174/
https://www.ncbi.nlm.nih.gov/pubmed/32117013
http://dx.doi.org/10.3389/fneur.2020.00045

Ejemplares similares