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risdiplam (Evrysdi)
Indications:
- treatment of spinal muscular atrophy
Contraindications: -pregnancy
Dosage:
- 5 mg PO QD
- 0.2 mg/kg if < 2 years of age
60 mg of risdiplam powder for constitution to provide 0.75 mg/mL solution
Adverse effects:
- fever, diarrhea, rash (> 10%)
Mechanism of action:
- survival of motor neuron 2 (SMN2) splicing modifier
General
heterocyclic compound, 2 rings
neurologic agent
Database Correlations
PUBCHEM cid=118513932
References
- Johnson K
Risdiplam Promising for Spinal Muscular Atrophy.
Medscape - Mar 09, 2021.
https://www.medscape.com/viewarticle/947139
- Baranello G, Darras BT, Day JW et al
Risdiplam in Type 1 Spinal Muscular Atrophy
N Engl J Med 2021; 384:915-923
PMID: 33626251
https://www.nejm.org/doi/full/10.1056/NEJMoa2009965
- Highlights of prescribing information
Evrysdi (risdiplam) for oral solution
https://www.gene.com/download/pdf/evrysdi_prescribing.pdf