Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries

Background: Nusinersen is an orphan drug intended for the treatment of spinal muscular atrophy (SMA), a severe genetic neuromuscular disorder. Considering the very high costs of orphan drugs and the expected market entry of cell and gene therapies, there is increased interest in the use of health te...

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Detalles Bibliográficos
Autores principales: Blonda, Alessandra, Barcina Lacosta, Teresa, Toumi, Mondher, Simoens, Steven
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Pharmacology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8814578/
https://www.ncbi.nlm.nih.gov/pubmed/35126102
http://dx.doi.org/10.3389/fphar.2021.750742

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8814578/
https://www.ncbi.nlm.nih.gov/pubmed/35126102
http://dx.doi.org/10.3389/fphar.2021.750742

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