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Year Number of Results
2018 5
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2020 21
2021 38
2022 57
2023 63
2024 30

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Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data.
Erdos J, Wild C. Erdos J, et al. Eur J Paediatr Neurol. 2022 Jul;39:1-10. doi: 10.1016/j.ejpn.2022.04.006. Epub 2022 Apr 30. Eur J Paediatr Neurol. 2022. PMID: 35533607 Free article. Review.
Drug-related adverse events occurred rarely in all types of SMA patients with all assessed drugs. Mid- and long-term studies on risdiplam could not be identified. CONCLUSIONS: The large quantity of missing data and heterogeneity of studies hinder comparability. ...
Drug-related adverse events occurred rarely in all types of SMA patients with all assessed drugs. Mid- and long-term studies on risdiplam …
Risdiplam: A Review in Spinal Muscular Atrophy.
Paik J. Paik J. CNS Drugs. 2022 Apr;36(4):401-410. doi: 10.1007/s40263-022-00910-8. Epub 2022 Mar 13. CNS Drugs. 2022. PMID: 35284988 Review.
These motor improvements were maintained with up to 2 years of treatment with risdiplam. Risdiplam was generally well tolerated, with a favourable benefit to risk balance. ...Risdiplam was generally well tolerated in these trials, with a favourable benefit to …
These motor improvements were maintained with up to 2 years of treatment with risdiplam. Risdiplam was generally well tolerate …
Curing SMA: Are we there yet?
Reilly A, Chehade L, Kothary R. Reilly A, et al. Gene Ther. 2023 Feb;30(1-2):8-17. doi: 10.1038/s41434-022-00349-y. Epub 2022 May 26. Gene Ther. 2023. PMID: 35614235 Review.
With three relatively new available therapies, nusinersen (Spinraza), onasemnogene abeparvovec (Zolgensma), and risdiplam (Evrysdi), patients survive longer and have improved outcomes. ...
With three relatively new available therapies, nusinersen (Spinraza), onasemnogene abeparvovec (Zolgensma), and risdiplam (Evrysdi), …
[Update on spinal muscular atrophy treatment].
Erazo Torricelli R. Erazo Torricelli R. Medicina (B Aires). 2022 Aug 30;82 Suppl 3:76-81. Medicina (B Aires). 2022. PMID: 36054863 Free article. Review. Spanish.
Currently, the existence of genetic treatments: gene modification by inclusion of exon 7 to the SMN2 gene (nusinersen and risdiplam) or insertion of the SMN1 gene through the adeno-associated viral transporter (onasemnogene) have radically modified the clinical evolution o …
Currently, the existence of genetic treatments: gene modification by inclusion of exon 7 to the SMN2 gene (nusinersen and risdiplam) …
Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA).
Oskoui M, Day JW, Deconinck N, Mazzone ES, Nascimento A, Saito K, Vuillerot C, Baranello G, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Papp G, Gorni K, Kletzl H, Martin C, McIver T, Scalco RS, Staunton H, Yeung WY, Fontoura P, Mercuri E; SUNFISH Working Group. Oskoui M, et al. J Neurol. 2023 May;270(5):2531-2546. doi: 10.1007/s00415-023-11560-1. Epub 2023 Feb 3. J Neurol. 2023. PMID: 36735057 Free PMC article. Clinical Trial.
Risdiplam is an oral, survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier approved for the treatment of spinal muscular atrophy (SMA). ...After 12 months, all participants received risdiplam while preserving initial treatment blinding. We report 24-month ef …
Risdiplam is an oral, survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier approved for the treatment of spinal muscular atro …
Spinal Muscular Atrophy.
Nicolau S, Waldrop MA, Connolly AM, Mendell JR. Nicolau S, et al. Semin Pediatr Neurol. 2021 Apr;37:100878. doi: 10.1016/j.spen.2021.100878. Epub 2021 Feb 11. Semin Pediatr Neurol. 2021. PMID: 33892848 Review.
Three different disease-modifying treatments were introduced in the last 4 years: nusinersen, onasemnogene abeparvovec, and risdiplam. These agents have demonstrated safety and efficacy, but their long-term benefits require further study. ...
Three different disease-modifying treatments were introduced in the last 4 years: nusinersen, onasemnogene abeparvovec, and risdiplam …
Targeting RNA structures with small molecules.
Childs-Disney JL, Yang X, Gibaut QMR, Tong Y, Batey RT, Disney MD. Childs-Disney JL, et al. Nat Rev Drug Discov. 2022 Oct;21(10):736-762. doi: 10.1038/s41573-022-00521-4. Epub 2022 Aug 8. Nat Rev Drug Discov. 2022. PMID: 35941229 Free PMC article. Review.
Existing RNA-targeted small molecules use a range of mechanisms including directing splicing - by acting as molecular glues with cellular proteins (such as branaplam and the FDA-approved risdiplam), inhibition of translation of undruggable proteins and deactivation of func …
Existing RNA-targeted small molecules use a range of mechanisms including directing splicing - by acting as molecular glues with cellular pr …
How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison.
Ribero VA, Daigl M, Martí Y, Gorni K, Evans R, Scott DA, Mahajan A, Abrams KR, Hawkins N. Ribero VA, et al. J Comp Eff Res. 2022 Apr;11(5):347-370. doi: 10.2217/cer-2021-0216. Epub 2022 Jan 18. J Comp Eff Res. 2022. PMID: 35040693 Free article. Review.
Aim: To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). ...Results: In Type 1 SMA, studies of risdiplam and nusinersen included similar populations. Indirect comparison results found improved …
Aim: To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). ... …
Spinal Muscular Atrophy.
Oskoui M, Servais L. Oskoui M, et al. Continuum (Minneap Minn). 2023 Oct 1;29(5):1564-1584. doi: 10.1212/CON.0000000000001338. Continuum (Minneap Minn). 2023. PMID: 37851043 Review.
Pharmacologic treatments approved by the US Food and Drug Administration (FDA) include three drugs that increase deficient survival motor neuron protein levels through SMN1 - or SMN2 - directed pathways: nusinersen, onasemnogene abeparvovec, and risdiplam. Efficacy for the …
Pharmacologic treatments approved by the US Food and Drug Administration (FDA) include three drugs that increase deficient survival motor ne …
192 results