DEVOTE Study Exploring Higher Dose of Nusinersen in Spinal Muscular Atrophy: Study Design and Part A Results

BACKGROUND: Pharmacokinetic/pharmacodynamic modeling indicates that the higher dose of nusinersen may be associated with a clinically meaningful increase in efficacy above that seen with the 12-mg approved dose. OBJECTIVE: Here we describe both the design of DEVOTE (NCT04089566), a 3-part clinical s...

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Detalles Bibliográficos
Autores principales: Finkel, Richard S., Day, John W., Pascual Pascual, Samuel Ignacio, Ryan, Monique M., Mercuri, Eugenio, De Vivo, Darryl C., Montes, Jacqueline, Gurgel-Giannetti, Juliana, Monine, Michael, Gambino, Giulia, Makepeace, Corinne, Foster, Richard, Berger, Zdenek
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2023
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10578235/
https://www.ncbi.nlm.nih.gov/pubmed/37393513
http://dx.doi.org/10.3233/JND-221667

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10578235/
https://www.ncbi.nlm.nih.gov/pubmed/37393513
http://dx.doi.org/10.3233/JND-221667

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