Effect of Nusinersen in a late onset spinal muscular atrophy patient for 14 months: A case report

RATIONALE: Spinal muscular atrophy (SMA) is a genetic disorder caused by genetic defect of SMN1 gene. SMA was an untreatable disease until 2016, when Nusinersen an antisense oligonucleotide therapy was approved for treatment. We report the effect of Nusinersen in a late onset SMA for 14 months. PATI...

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Detalles Bibliográficos
Autores principales: Park, Jin-Mo, Min, Yu-Sun, Park, Donghwi, Park, Jin-Sung
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2021
Materias:
5300
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7793442/
https://www.ncbi.nlm.nih.gov/pubmed/33429824
http://dx.doi.org/10.1097/MD.0000000000024236

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7793442/
https://www.ncbi.nlm.nih.gov/pubmed/33429824
http://dx.doi.org/10.1097/MD.0000000000024236

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