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

• Egile Nagusiak: Alessandra Blonda (Egilea), Teresa Barcina Lacosta (Egilea), Mondher Toumi (Egilea), Steven Simoens (Egilea)
• Formatua: Liburua
• Argitaratua: Frontiers Media S.A., 2022-01-01T00:00:00Z.
• Gaiak: article
• Sarrera elektronikoa: Connect to this object online.

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...