Real World Evidence on the Effectiveness of Nusinersen within the National Program to Treat Spinal Muscular Atrophy in Poland

Background: Spinal muscular atrophy (SMA) is a debilitating neuromuscular disease resulting in children’s mortality and disability. Nusinersen is available to all SMA patients in Poland since 2019. Aim: To compare mortality or disease progression to mechanical ventilation in two patient cohorts befo...

Descripción completa

Detalles Bibliográficos
Autores principales: Krupa, Dominika, Czech, Marcin, Chudzyńska, Ewa, Koń, Beata, Kostera-Pruszczyk, Anna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10218344/
https://www.ncbi.nlm.nih.gov/pubmed/37239800
http://dx.doi.org/10.3390/healthcare11101515

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10218344/
https://www.ncbi.nlm.nih.gov/pubmed/37239800
http://dx.doi.org/10.3390/healthcare11101515

Ejemplares similares