The potential value of disease-modifying therapy in patients with spinocerebellar ataxia type 1: an early health economic modeling study

OBJECTIVE: There currently is no disease-modifying therapy for spinocerebellar ataxia type 1 (SCA1). Genetic interventions, such as RNA-based therapies, are being developed but those currently available are very expensive. Early evaluation of costs and benefits is, therefore, crucial. By developing...

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Detalles Bibliográficos
Autores principales: van Prooije, Teije, Ruigrok, Sanne, van den Berkmortel, Niels, Maas, Roderick P. P. W. M., Wijn, Stan, van Roon-Mom, Willeke M. C., van de Warrenburg, Bart, Grutters, Janneke P. C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2023
Materias:
Original Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10344992/
https://www.ncbi.nlm.nih.gov/pubmed/37076599
http://dx.doi.org/10.1007/s00415-023-11704-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10344992/
https://www.ncbi.nlm.nih.gov/pubmed/37076599
http://dx.doi.org/10.1007/s00415-023-11704-3

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