Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive, neurodegenerative disease caused by biallelic mutations in the survival motor neuron 1 (SMN1) gene. SMA is characterized by motor neuron degeneration, resulting in progressive muscle atrophy and weakness. Before the emergence of disease‐modify...

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Detalles Bibliográficos
Autores principales: Kichula, Elizabeth A., Proud, Crystal M., Farrar, Michelle A., Kwon, Jennifer M., Saito, Kayoko, Desguerre, Isabelle, McMillan, Hugh J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2021
Materias:
Issues & Opinions
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8518380/
https://www.ncbi.nlm.nih.gov/pubmed/34196026
http://dx.doi.org/10.1002/mus.27363

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8518380/
https://www.ncbi.nlm.nih.gov/pubmed/34196026
http://dx.doi.org/10.1002/mus.27363

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