Disease Burden of Spinal Muscular Atrophy: A Comparative Cohort Study Using Insurance Claims Data in the USA

BACKGROUND: Spinal muscular atrophy (SMA) is a neuromuscular disease caused by homozygous deletion or loss-of-function mutations of the survival of motor neuron 1 (SMN1) gene, resulting in reduced levels of SMN protein throughout the body. Patients with SMA may have multiple tissue defects, which co...

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Detalles Bibliográficos
Autores principales: Mouchet, Julie, Roumpanis, Spyros, Gaki, Eleni, Lipnick, Scott, Oskoui, Maryam, Scalco, Renata S., Darras, Basil T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2023
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9881018/
https://www.ncbi.nlm.nih.gov/pubmed/36314213
http://dx.doi.org/10.3233/JND-210764

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