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Effectiveness of Nusinersen in Type 1, 2 and 3 Spinal Muscular Atrophy: Croatian Real-World Data

(1) Background: To investigate the real-world effectiveness and safety profile of nusinersen in Croatian paediatric and adult spinal muscular atrophy (SMA) patients. (2) Methods: A retrospective and anonymous collection of relevant demographic and clinical data for all Croatian SMA patients treated...

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Detalles Bibliográficos
Autores principales: Belančić, Andrej, Strbad, Tea, Kučan Štiglić, Marta, Vitezić, Dinko
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10142582/
https://www.ncbi.nlm.nih.gov/pubmed/37109175
http://dx.doi.org/10.3390/jcm12082839