Cost effectiveness of nusinersen for patients with infantile-onset spinal muscular atrophy in US

BACKGROUND: Patients with infantile-onset spinal muscular atrophy (SMA), a rare, genetic neuromuscular disease, do not achieve key motor function milestones (e.g., sitting) and have short life expectancy in the absence of treatment. Nusinersen is a disease-modifying therapy for patients with SMA. OB...

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Detalles Bibliográficos
Autores principales: Thokala, Praveen, Stevenson, Matt, Kumar, Varun M., Ren, Shijie, Ellis, Alexandra G., Chapman, Richard H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2020
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7539471/
https://www.ncbi.nlm.nih.gov/pubmed/33041673
http://dx.doi.org/10.1186/s12962-020-00234-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7539471/
https://www.ncbi.nlm.nih.gov/pubmed/33041673
http://dx.doi.org/10.1186/s12962-020-00234-8

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