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Poster Communications Themes

Theme 09 - Clinical Trials and Trial Design

Reference

  • Liu Y, Dodart JC, Tran H, et al. Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models. Nature communications. 2021;12(1):847.

References

  • Klim JR, Williams LA, Limone F, et al. ALS-implicated protein TDP-43 sustains levels of STMN2, a mediator of motor neuron growth and repair. Nat Neurosci. 2019;22(2):167–79.
  • Melamed Z, López-Erauskin J, Baughn MW, et al. Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration. Nat Neurosci. 2019;22(2):180–90.

References

  • Elia A, Lalli S, Monsurrò M, et al. Tauroursodeoxycholic acid in the treatment of patients with amyotrophic lateral sclerosis. Eur J Neurol. 2016;23(1):45–52.
  • Paganoni S, Macklin E, Hendrix S, et al. Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis. N Engl J Med. 2020;383(10):919–30.
  • Tornese P, Lalli S, Cocco A, et al. Review of disease-modifying drug trials in amyotrophic lateral sclerosis. J Neurol Neurosurg Psychiatry. 2022;93(5):521–9.

References

  • Dudley L, Gamble C, Preston J, et al. What Difference Does Patient and Public Involvement Make and What Are Its Pathways to Impact? Qualitative Study of Patients and Researchers from a Cohort of Randomised Clinical Trials. PLOS One. 2015;8:e0128817.
  • Ochieng CA, Minion JT, Turner A, et al. What does engagement mean to participants in longitudinal cohort studies? A qualitative study. BMC Med Ethics. 2021;22:77.
  • Bedlack R, Pogemiller A, Shefner J, et al. ALS clinical research learning institutes (ALS-CRLI): empowering people with ALS to be research ambassadors. Amyotroph Lateral Scler Frontotemporal Degener. 2020;21:216–221.
  • Picher-Martel V, Magnussen C, Mathieu M, et al. CAPTURE ALS: the comprehensive analysis platform to understand, remedy and eliminate ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2022;1–7.

References

  • Morgan DL. 2018. Basic and advanced focus groups. SAGE Publications.
  • Davies L, LeClair KL, Bagley P, et al. Face-to-Face Compared With Online Collected Accounts of Health and Illness Experiences: A Scoping Review. Qual Health Res. 2020;30(13):2092–102.
  • Genuis SK, Luth W, Bubela T, Johnston WS. Covid-19 threat and coping: application of protection motivation theory to the pandemic experiences of people affected by amyotrophic lateral sclerosis. BMC Neurol. 2022;22(140).

References

  • Shefner JM, Andrews JA, Genge A, et al. A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2021;22:287–299.
  • Westeneng H-J, Debray TPA, Vissar AE, et al. Prognosis for patients with amyotrophic lateral sclerosis: development and validation of a personalised prediction model. Lancet Neurol. 2018;17:423–433.

Reference

  • Abidi M, de Marco G, Grami F, et al. Neural Correlates of Motor Imagery of Gait in Amyotrophic Lateral Sclerosis. J Magn Reson Imaging. 2021;53(1):223–233.

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