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Switching from Nusinersen to Risdiplam: A Croatian Real-World Experience on Effectiveness and Safety

Journal of Personalized Medicine
: (1) Background: Data on combination or sequential treatment of spinal muscular atrophy (SMA) with disease-modifying drugs (DMDs) are missing and the latter field is poorly understood.
Andrej Belančić, Dinko Vitezic, Marta Kucan +2 more
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muscular atrophy, spinal
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Risdiplam: First Approval

Drugs, 2020
Risdiplam (Evrysdi™) is an orally administered, survival motor neuron 2 (SMN2)-directed RNA splicing modifier being developed by Roche, PTC Therapeutics Inc and the SMA Foundation for the treatment of the spinal muscular atrophy. The small molecule is designed to treat spinal muscular atrophy caused by mutations in chromosome 5q leading to SMN protein ...
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Risdiplam for the treatment of spinal muscular atrophy

S.S. Korsakov Journal of Neurology and Psychiatry
Spinal muscular atrophy (SMA) is a devastating disease that is the leading genetic cause of death in infants and young children. It includes a broad spectrum of phenotypes that are classified into clinical groups based on the age of onset and maximum motor function achieved.
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Risdiplam

Reactions Weekly
+5 more sources

Risdiplam

Reactions Weekly, 2023
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Nusinersen/risdiplam

Reactions Weekly, 2023
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Risdiplam: new opportunities but more to be done

The Lancet Neurology, 2022
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Zulassungserweiterung für Risdiplam

DNP – Die Neurologie & Psychiatrie, 2023
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Children With Spinal Muscular Atrophy Treated With Nusinersen/Risdiplam