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Shpilyukova Yu.A.

Research Center of Neurology

Illarioshkin S.N.

Research Center of Neurology

Oral disease-modifying therapy for adult patients with spinal muscular atrophy type 2

Authors:

Shpilyukova Yu.A., Illarioshkin S.N.

More about the authors

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To cite this article:

Shpilyukova YuA, Illarioshkin SN. Oral disease-modifying therapy for adult patients with spinal muscular atrophy type 2. S.S. Korsakov Journal of Neurology and Psychiatry. 2023;123(12):148‑153. (In Russ., In Engl.)
https://doi.org/10.17116/jnevro2023123121148

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References:

  1. Wan H, Carey C, D’Silva A, et al. Health, wellbeing and lived experiences of adults with SMA: a scoping systematic review. Orphanet J Rare Dis. 2020;15(70):1-17.  https://doi.org/10.1186/s13023-020-1339-3
  2. Rad N, Cai H, Weiss MD. Management of Spinal Muscular Atrophy in the Adult Population. Muscle Nerve. 2022;(February):498-507.  https://doi.org/10.1002/mus.27519
  3. Hagenacker T, Wurster CD, Günther R, et al. Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study. Lancet Neurol. 2020;19(4):317-325.  https://doi.org/10.1016/S1474-4422(20)30037-5
  4. Maggi L, Bello L, Bonanno S, et al. Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3. J Neurol Neurosurg Psychiatry. 2020;91(11):1166-1174. https://doi.org/10.1136/jnnp-2020-323822
  5. Mercuri E, Deconinck N, Mazzone ES, et al. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2022;21(1):42-52.  https://doi.org/10.1016/S1474-4422(21)00367-7
  6. Mercuri E, Baranello G, Boespflug-Tanguy O, et al. Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment. Eur J Neurol. 2023;30:1945-1956. https://doi.org/10.1111/ene.15499
  7. Novikova ES. Oral risdiplam for specific therapy in adult patients with 5q spinal muscular atrophy in the Moscow region. Ann Clin Exp Neurol. 2023;17(3):88-93. (In Russ.). https://doi.org/10.54101/ACEN.2023.3.11
  8. McCluskey G, Lamb S, Mason S, et al. Risdiplam for the treatment of adults with spinal muscular atrophy: Experience of the Northern Ireland neuromuscular service. Muscle Nerve. 2023;67(2):157-161.  https://doi.org/10.1002/mus.27755
  9. Ñungo Garzón NC, Pitarch Castellano I, Sevilla T, Vázquez‐Costa JF. Risdiplam in non‐sitter patients aged 16 years and older with 5q spinal muscular atrophy. Muscle Nerve. 2023;67(5):407-411.  https://doi.org/10.1002/mus.27804
  10. Schreiber-Katz O, Siegler HA, Wieselmann G, et al. Improvement of muscle strength in specific muscular regions in nusinersen-treated adult patients with 5q-spinal muscular atrophy. Sci Rep. 2023;13(1):6240. https://doi.org/10.1038/s41598-023-31617-5

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