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...

Täydet tiedot

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Bibliografiset tiedot
Julkaisussa:Ann Clin Transl Neurol
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: John Wiley and Sons Inc. 2020
Aiheet:
Research Articles
Linkit: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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