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. 2024 Oct 31;65(5):454-456.
doi: 10.3325/cmj.2024.65.454.

Could choosing risdiplam instead of nusinersen in the treatment of type 1 spinal muscular atrophy be a huge cost-minimization opportunity?

Andrej Belančić  1 , Andrea Katrin Faour, Elvira Meni Maria Gkrinia, Dinko Vitezić
Affiliations

Could choosing risdiplam instead of nusinersen in the treatment of type 1 spinal muscular atrophy be a huge cost-minimization opportunity?

Andrej Belančić et al. Croat Med J. .
No abstract available

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References

    1. Yeo CJJ, Tizzano EF, Darras BT. Challenges and opportunities in spinal muscular atrophy therapeutics. Lancet Neurol. 2024;23:205–18. doi: 10.1016/S1474-4422(23)00419-2. - DOI - PubMed
    1. Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377:1723–32. doi: 10.1056/NEJMoa1702752. - DOI - PubMed
    1. Mercuri E, Muntoni F, Baranello G, Masson R, Boespflug-Tanguy O, Bruno C, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021;20:832–41. doi: 10.1016/S1474-4422(21)00251-9. - DOI - PubMed
    1. Darras BT, Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Xiong H, Zanoteli E, et al. Risdiplam-treated infants with type 1 spinal muscular atrophy versus historical controls. N Engl J Med. 2021;385:427–35. doi: 10.1056/NEJMoa2102047. - DOI - PubMed
    1. Ribero VA, Daigl M, Martí Y, Gorni K, Evans R, Scott DA, et al. How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison. J Comp Eff Res. 2022;11:347–70. doi: 10.2217/cer-2021-0216. - DOI - PubMed

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