Cited by 50

The DOI matches its Crossref record; 2 of the 2 references it cites were found in a registry or an index.

Nusinersen: A Review in 5q Spinal Muscular Atrophy

Hoy, Sheridan M.·2021·CNS Drugs

Overview

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

A few free checks a day · papers already verified open instantly · results are public

Integrity signals

Registry

Crossref · 10.1007/s40263-021-00878-x

Cites

2 of 2 references verified0 citation errors · 0 not checkable

Statistics

Verification

Claims the paper makes

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

A few free checks a day · papers already verified open instantly · results are public

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

Self-check

Percentages, sums and reported differences re-computed from the paper’s own numbers.

The self-check needs the paper’s full text.

Citations

Cites

Show all 2 references

Research works printed but not linked: 53

  1. Groen EJN, Talbot K, Gillingwater TH. Advances in therapy for spinal muscular atrophy: promises and challenges. Nat Rev Neurol. 2018;14(4):214–24. · DOI
  2. Brakemeier S, Stolte B, Thimm A, et al. Assessment of bulbar function in adult patients with 5q-SMA Type 2 and 3 under treatment with nusinersen. Brain Sci. 2021;11(9):1244. · DOI
  3. Wadman RI, van der Pol WL, Bosboom WM, et al. Drug treatment for spinal muscular atrophy type I. Cochrane Database Syst Rev. 2019;12(Cd006281):1–46.
  4. Wadman RI, van der Pol WL, Bosboom WM, et al. Drug treatment for spinal muscular atrophy types II and III. Cochrane Database Syst Rev. 2020;1(Cd006282):1–97.
  5. Volpe JJ. Infantile spinal muscular atrophy: the potential for cure of a fatal disease. J Neonatal Perinatal Med. 2021;14(2):153–7. · DOI
  6. Hoy SM. Nusinersen: a review in 5q spinal muscular atrophy. CNS Drugs. 2018;32(7):689–96. · DOI
  7. De Vivo DC, Bertini E, Swoboda KJ, et al. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: interim efficacy and safety results from the phase 2 NURTURE study. Neuromuscul Disord. 2019;29(11):842–56. · DOI
  8. Swoboda KJ, Sansone VA, De Vivo DC, et al. Preserved swallowing function in infants who initiated nusinersen treatment in the presymptomatic stage of SMA: results from the NURTURE study [abstract]. In: 2021 MDA Virtual Clinical & Scientific Conference. 2021.
  9. Finkel RS, Chiriboga CA, Vajsar J, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016;388(10063):3017–26. · DOI
  10. Kuntz N, Tizzano E, Topaloglu H, et al. Time to motor function response among nusinersen-treated infants from the ENDEAR study [abstract no. 141]. Ann Neurol. 2018;84(Suppl 22):S389–90.
  11. Finkel R, Kirschner J, Mercuri E, et al. Longer-term effects of nusinersen on motor function outcomes based on age at treatment initiation [abstract no P.264]. Neuromuscul Disord. 2020;30(Suppl 1):S123–4. · DOI
  12. Servais L, Finkel RS, Kirschner J, et al. Longer-term effects of nusinersen on motor function outcomes based on age at treatment initiation [abstract no. 29]. Dev Med Child Neurol. 2021;63(Suppl 1):23.
  13. Servais L, Finkel RS, Castro D, et al. Predictive factors of nusinersen treatment response in infantile-onset spinal muscular atrophy: results from the ENDEAR/SHINE studies [abstract]. In: 2021 MDA Virtual Clinical & Scientific Conference. 2021.
  14. Pane M, Palermo C, Messina S, et al. Nusinersen in type 1 SMA infants, children and young adults: preliminary results on motor function. Neuromuscul Disord. 2018;28(7):582–5. · DOI
  15. Messina S, Sframeli M, Bruno C, et al. Nutritional status in SMA type 1 over a 6 month follow-up in nusinersen expanded access program (EAP) [abstract]. Acta Myol. 2018;37(1):53–4.
  16. Pane M, Coratti G, Sansone VA, et al. Nusinersen in type 1 spinal muscular atrophy: twelve-month real-world data. Ann Neurol. 2019;86(3):443–51. · DOI
  17. Pane M, Coratti G, Sansone VA, et al. Type I SMA “new natural history”: long-term data in nusinersen-treated patients. Ann Clin Transl Neurol. 2021;8(3):548–57. · DOI
  18. Mercuri E, Darras BT, Chiriboga CA, et al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018;378(7):625–35. · DOI · Paper page
  19. Mercuri E, Darras BT, Chiriboga CA, et al. Longer-term treatment with nusinersen: results in later-onset spinal muscular atrophy from the SHINE study [abstract no. 265]. J Neuromuscular Dis. 2021;8(S1):S67.
  20. Darras BT, Chiriboga CA, Iannaccone ST, et al. Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1/2 studies. Neurology. 2019;92(21):e2492–506. · DOI
  21. Coratti G, Pane M, Lucibello S, et al. Age related treatment effect in type II spinal muscular atrophy pediatric patients treated with nusinersen. Neuromuscul Disord. 2021;31(7):596–602. · DOI
  22. Pera MC, Coratti G, Bovis F, et al. Nusinersen in pediatric and adult patients with type III spinal muscular atrophy. Ann Clin Transl Neurol. 2021;8(8):1622–34. · DOI
  23. 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):317–25. · DOI
  24. 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):1166–74. · DOI
  25. Duong T, Wolford C, McDermott MP, et al. Nusinersen treatment in adults with spinal muscular atrophy. Neurol Clin Pract. 2021;11(3):e317–27. · DOI
  26. Elsheikh B, Severyn S, Zhao S, et al. Safety, tolerability, and effect of nusinersen treatment in ambulatory adults with 5q-SMA. Front Neurol. 2021;12:650635.
  27. Elsheikh B, Severyn S, Zhao S, et al. Safety, tolerability, and effect of nusinersen in non-ambulatory adults with spinal muscular atrophy. Front Neurol. 2021;12:65032.
  28. Osmanovic A, Ranxha G, Kumpe M, et al. Treatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy. Ther Adv Neurol Disord. 2021;14:1756286421998902. · DOI
  29. Acsadi G, Crawford TO, Muller-Felber W, et al. Safety and efficacy of nusinersen in spinal muscular atrophy: the EMBRACE study. Muscle Nerve. 2021;63:668–77. · DOI
  30. Audic F, de la Banda MGG, Bernoux D, et al. Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study. Orphanet J Rare Dis. 2020;15(148):1–10.
  31. Darras BT, Farrar MA, Mercuri E, et al. An integrated safety analysis of infants and children with symptomatic spinal muscular atrophy (SMA) treated with nusinersen in seven clinical trials. CNS Drugs. 2019;33(9):919–32. · DOI
  32. Farrar M, Castro D, Finkel R, et al. Longer-term safety data in individuals with later-onset SMA support the favourable tolerability profile of nusinersen [abstract no. EPR-123]. Eur J Neurol. 2021;28(Suppl 1):287.
  33. Stolte B, Nonnemacher M, Kizina K, et al. Nusinersen treatment in adult patients with spinal muscular atrophy: a safety analysis of laboratory parameters. J Neurol. 2021;268(12):4667–79. · DOI
  34. Sansone V, Finkel R, Tulinius M, et al. Integrated analysis of annualized incidence of serious adverse events in infantile-onset SMA treated with nusinersen [abstract no. 765]. J Neuromuscular Dis. 2021;8(Suppl 1):S152–3.
  35. Dhillon S. Risdiplam: first approval. Drugs. 2020;80(17):1853–8. · DOI
  36. Messina S, Sframeli M. New treatments in spinal muscular atrophy: positive results and new challenges. J Clin Med. 2020;9(7):13. · DOI
  37. Mercuri E, Sansone V. Nusinersen in adults with spinal muscular atrophy: new challenges. Lancet Neurol. 2020;19(4):283–4. · DOI
  38. Weststrate H, Stimpson G, Thomas L, et al. Bulbar function evolution in SMA type 1 children treated with nusinersen [abstract no. EP.276]. Neuromuscul Disord. 2021;31(Suppl 1):S133–4. · DOI
  39. LoMauro A, Mastella C, Alberti K, et al. Effect of nusinersen on respiratory muscle function in different subtypes of type 1 spinal muscular atrophy. Am J Respir Crit Care Med. 2019;200(12):1547–50. · DOI
  40. Chacko A, Sly PD, Ware RS, et al. Effect of nusinersen on respiratory function in paediatric spinal muscular atrophy types 1-3. Thorax. 2021. https://doi.org/10.1136/thoraxjnl-2020-216564. · DOI
  41. Viscidi E, Wang N, Juneja M, et al. The incidence of hydrocephalus among patients with and without spinal muscular atrophy (SMA): results from a US electronic health records study. Orphanet J Rare Dis. 2021;16(1):207. · DOI
  42. Dangouloff T, Botty C, Beaudart C, et al. Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments. Orphanet J Rare Dis. 2021;16(1):47. · DOI
  43. Pearson SD, Thokala P, Stevenson M, et al. The effectiveness and value of treatments for spinal muscular atrophy: a summary from the Institute for Clinical and Economic Review’s New England Comparative Effectiveness Public Advisory Council. J Manag Care Spec Pharm. 2019;25(12):1300–6.
  44. Dean R, Jensen I, Cyr P, et al. An updated cost-utility model for onasemnogene abeparvovec (Zolgensma(R)) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER). J Mark Access Health Policy. 2021;9(1):1889841. · DOI
  45. Broekhoff TF, Sweegers CCG, Krijkamp EM, et al. Early cost-effectiveness of onasemnogene abeparvovec-xioi (Zolgensma) and nusinersen (Spinraza) treatment for spinal muscular atrophy I in the Netherlands with relapse scenarios. Value Health. 2021;24(6):759–69. · DOI
  46. Ollendorf DA, Chapman RH, Pearson SD. Evaluating and valuing drugs for rare conditions: no easy answers. Value Health. 2018;21(5):547–52. · DOI
  47. Darras BT, Crawford TO, Finkel RS, et al. Neurofilament as a potential biomarker for spinal muscular atrophy. Ann Clin Transl Neurol. 2019;6(5):932–44. · DOI
  48. Finkel R, Sumner C, Darras B, et al. Association between plasma phosphorylated neurofilament heavy chain and efficacy endpoints in the nusinersen NURTURE study [abstract no. 173]. Ann Neurol. 2019;86(Suppl 23):S122.
  49. Sumner CJ, Darras BT, Muntoni F, et al. Association of phosphorylated neurofilament heavy chain (pNF-H) levels with motor function achievement in individuals with spinal muscular atrophy (SMA) treated with nusinersen [abstract no. S27.009]. Neurology. 2019;92(15 Suppl 1). · DOI
  50. Darras BT, Sumner CJ, Muntoni F, et al. Plasma phosphorylated neurofilament heavy chain levels over time in participants with infantile-and later-onset SMA: data from the SHINE study [abstract no. 1692]. Neurology. 2020;94(15 Suppl). · DOI
  51. Bonanno S, Marcuzzo S, Malacarne C, et al. Circulating myomiRs as potential biomarkers to monitor response to nusinersen in pediatric SMA patients. Biomedicines. 2020;8(2):21. · DOI
  52. Gómez-Garcia de la Banda M, Amaddeo A, Khirani S, et al. Assessment of respiratory muscles and motor function in children with SMA treated by nusinersen. Pediatr Pulmonol. 2020;56(1):299–306. · DOI
  53. Sansone VA, Pirola A, Albamonte E, et al. Respiratory needs in patients with type 1 spinal muscular atrophy treated with nusinersen. J Pediatr. 2020;219:223-8.e4. · DOI

Cited by

Sorted by citations

See all 50

Metadata

Authors
Hoy, S. M.
Venue
CNS Drugs
Published
2021-11-30
Volume
35
Issue
12
Pages
1317-1328
ISSN
  • 1172-7047
  • 1179-1934
Cited by
50
References
2
Language
en

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review

How to cite

Cite CoChat for the checks on this page, and the paper for its own findings.

This page
CoChat. (2026). Citation check: Nusinersen: A Review in 5q Spinal Muscular Atrophy. https://app.cochat.ai/papers/10.1007/s40263-021-00878-x
The paper
Hoy, S. M. (2021). Nusinersen: A Review in 5q Spinal Muscular Atrophy. CNS Drugs. https://doi.org/10.1007/s40263-021-00878-x