Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophy

OBJECTIVE: Evaluation of ophthalmologic safety with focus on retinal safety in patients with spinal muscular atrophy (SMA) treated with risdiplam (EVRYSDI®), a survival of motor neuron 2 splicing modifier associated with retinal toxicity in monkeys. Risdiplam was approved recently for the treatment...

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Detalles Bibliográficos
Autores principales: Sergott, Robert C., Amorelli, Giulia M., Baranello, Giovanni, Barreau, Emmanuel, Beres, Shannon, Kane, Steven, Mercuri, Eugenio, Orazi, Lorenzo, SantaMaria, Melissa, Tremolada, Gemma, Santarsiero, Diletta, Waskowska, Agnieszka, Yashiro, Shigeko, Denk, Nora, Fürst‐Recktenwald, Sabine, Gerber, Marianne, Gorni, Ksenija, Jaber, Birgit, Jacobsen, Bjoern, Mueller, Lutz, Nave, Stephane, Scalco, Renata S., Marzoli, Stefania B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7818230/
https://www.ncbi.nlm.nih.gov/pubmed/33231373
http://dx.doi.org/10.1002/acn3.51239

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