Metabolic Dysfunction in Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive genetic disorder leading to paralysis, muscle atrophy, and death. Significant advances in antisense oligonucleotide treatment and gene therapy have made it possible for SMA patients to benefit from improvements in many aspects of the once devas...

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Detalles Bibliográficos
Autores principales: Deguise, Marc-Olivier, Chehade, Lucia, Kothary, Rashmi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8198411/
https://www.ncbi.nlm.nih.gov/pubmed/34072857
http://dx.doi.org/10.3390/ijms22115913

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