DOI : 10.1055/s-00000041

Neuropediatrics

Issue S 01 · Volume 56 · October 2025 DOI: 10.1055/s-015-62321


NEP 50th Annual Meeting of the Society for Neuropediatrics, 2025
Bern, Switzerland, 02.–04.10.2025


  • A-235
    Küpper, H.; Roland-Schäfer, D.; Scheckenbach, C.; Kehrer, M.; Tizia, M.; Horber, V.; Rosewich, H.:

    “Warm-Up”-Like Phenomen in Andersen–Tawil-Syndrome: A Case Report and Comparative Analysis to Myotonica Congenita

  • A-172
  • A-273
  • A-132
    Veerapandiyan, A.; Eskuri, J.; Flanigan, K.; Laverty, C.; Phan, H.; Smith, E.; Rocha, C. Tesi; Wong, B.; Morris, M.; Tami, Y.; Carmack, T.; Kovach, P.; Herzog, J.; Hughes, S.; Zhu, Y.; Ackermann, E.:

    Del-Zota Produced Statistically Significant Increases in Exon Skipping and Dystrophin Levels in EXPLORE44: A Phase 1/2 Study in Patients with DMD44

  • A-134
    Werner, C.; Gruenert, J.; Mercuri, E.; Buccella, F.; Osorio, A. N.; Tulinius, M.; Desguerre, I.; de Resende, M. B. Dutra; McDonald, C.; Gordish-Dressman, H.; Morgenroth, L.; Johnson, S.; Krolick, A.; Anbu, B.; Liu, E.; Muntoni, F.:

    Effectiveness of Ataluren in Patients with nmDMD: Confirmatory Evidence from the STRIDE Registry

  • A-256
  • A-135
    Seelmann, I.; Mendell, J. R.; Muntoni, F.; McDonald, C. M.; Mercuri, E.; Ciafaloni, E.; Komaki, H.; Leon-Astudillo, C.; Nascimento, A.; Proud, C.; Schara-Schmidt, U.; Veerapandiyan, A.; Zaidman, C. M.; Furgerson, M.; Ding, K.; Singh, P.; Potter, R.; Asher, D. R.; Murphy, A. P.; Reid, C.; Hooper, G.; Torre, C. O.; Manfrini, M.; Elkins, J. S.; Rodino-Klapac, L. R.; Group, on behalf of the EMBARK Study:

    Long-Term Functional Outcomes, Safety, and Micro-Dystrophin Expression Following Delandistrogene Moxeparvovec Treatment in DMD: EMBARK 2-Year Results

  • A-109
    Gilling, K.; Brandsema, J.; Acsadi, G.; Coceani, N.; Alessi, F.; Cazzaniga, S.; Bettica, P.; Finanger, E. L.:

    Long-Term Safety of Givinostat in Patients with Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

  • A-180
  • A-174
    Distelmaier, F.; Corral-Sarasa, J.; Jiménez-Sánchez, L.; Díaz-Casado, M. E.; Rohmann, M.; Seibt, A.; Herebian, D.; Smits, S.; Münch, J.; Mayatepek, E.; Breitkreutz, J.; Husain, R.; López-Herrador, S.; González-García, P.; López, L.:

    Preclinical and First-in-Human Evidence Supporting 4-Hydroxybenzoic Acid as a Treatment for COQ2-associated Mitochondrial Disease

  • A-139
    Hackmann, T.; Bill, M.; Niederberger, S.; Beilschmidt, L.; Voss, D.:

    The Societal Costs of Duchenne Muscular Dystrophy in Germany