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

Robert C SergottGiulia M AmorelliGiovanni BaranelloEmmanuel BarreauShannon BeresSteven KaneEugenio MercuriLorenzo OraziMelissa SantaMariaGemma TremoladaDiletta SantarsieroAgnieszka WaskowskaShigeko YashiroNora DenkSabine Fürst-RecktenwaldMarianne GerberKsenija GorniBirgit JaberBjoern JacobsenLutz MuellerStephane NaveRenata S ScalcoStefania B Marzolinull null
Published in: Annals of clinical and translational neurology (2020)
Extensive ophthalmologic monitoring conducted in studies in patients with SMA confirmed that risdiplam does not induce ophthalmologic toxicity in pediatric or adult patients with SMA at the therapeutic dose. These results suggest that safety ophthalmologic monitoring is not needed in patients receiving risdiplam, as also reflected in the United States Prescribing Information for risdiplam.
Keyphrases
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