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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...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2020
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| Materias: | |
| 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 |