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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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Ann Clin Transl Neurol
Prif Awduron: 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.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: John Wiley and Sons Inc. 2020
Pynciau:
Research Articles
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC7818230/
https://ncbi.nlm.nih.gov/pubmed/33231373
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.51239
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