Synonyms: compound 1 [PMID: 30044619] | Evrysdi® | RG-7916 | RG7916 | RO-7034067 | RO7034067
risdiplam is an approved drug (FDA (2020), EMA (2021))
Compound class:
Synthetic organic
Comment: Risdiplam is a first-in-class, orally bioavailable splicing modifier [3]. Like nusinersen, it is intended to improve the efficiency of SMN2 (HGNC ID 11118; Uniprot Q16637) gene transcription in patients with spinal muscular atrophy (SMA) [2].
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Molecular structure representations generated using Open Babel