The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is 1 of the leading causes of infant mortality. SMA is mostly caused by low levels of Survival Motor Neuron (SMN) protein due to deletion of or mutation in the SMN1 gene. Its nearly identical copy, SMN2, fails to compensate for the loss of SMN1 due to predominant skippi...

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Detalles Bibliográficos
Autores principales: Singh, Ravindra N, Ottesen, Eric W, Singh, Natalia N
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2020
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7691903/
https://www.ncbi.nlm.nih.gov/pubmed/33283185
http://dx.doi.org/10.1177/2633105520973985

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7691903/
https://www.ncbi.nlm.nih.gov/pubmed/33283185
http://dx.doi.org/10.1177/2633105520973985

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