Spinal Muscular Atrophy after Nusinersen Therapy: Improved Physiology in Pediatric Patients with No Significant Change in Urine, Serum, and Liquor 1H-NMR Metabolomes in Comparison to an Age-Matched, Healthy Cohort

Spinal muscular atrophy (SMA) is a genetically heterogeneous group of rare neuromuscular diseases and was until recently the most common genetic cause of death in children. The effects of 2-month nusinersen therapy on urine, serum, and liquor 1H-NMR metabolomes in SMA males and females were not expl...

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Detalles Bibliográficos
Autores principales: Deutsch, Leon, Osredkar, Damjan, Plavec, Janez, Stres, Blaž
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8065886/
https://www.ncbi.nlm.nih.gov/pubmed/33808177
http://dx.doi.org/10.3390/metabo11040206

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8065886/
https://www.ncbi.nlm.nih.gov/pubmed/33808177
http://dx.doi.org/10.3390/metabo11040206

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