Health economic evaluation of risdiplam in patients with spinal muscular atrophy

• Autori principali: А. S. Kolbin (Autore), А. А. Kurylev (Autore), Yu. Е. Balykina (Autore), М. А. Proskurin (Autore), S. А. Mishinova (Autore)
• Natura: Libro
• Pubblicazione: IRBIS LLC, 2021-10-01T00:00:00Z.
• Soggetti: article
• Accesso online: Connect to this object online.

Background. Spinal muscular atrophies (SMA) are clinically and genetically heterogenous inherited orphan diseases leading to progressive spinal motoneurons degeneration and loss of function. Risdiplam and nusinersen are both authorized in Russia for pathogenic SMA treatment and included in the list...