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Neurology(R) Neuroimmunology & Neuroinflammation|November 12, 2025
IgM Anti-Ganglioside Binding and Complement Activation in an iPSC-Derived Motor Neuron Model for Multifocal Motor NeuropathyDaniëlle Krijgsman, Kim Dijkxhoorn, Elisabeth de Zeeuw, et al.
Annals of Neurology|September 11, 2009
Randomized sequential trial of valproic acid in amyotrophic lateral sclerosisSanne Piepers, Jan H Veldink, Sonja W de Jong, et al.
Human Molecular Genetics|March 17, 2025
A de novo deletion underlying spinal muscular atrophy: implications for carrier testing and genetic counselingMaria M Zwartkruis, Mirjam S de Pagter, Demi Gommers, et al.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|June 15, 2024
2024 update: European consensus statement on gene therapy for spinal muscular atrophyJanbernd Kirschner, Günther Bernert, Nina Butoianu, et al.
Nature Medicine|December 8, 2025
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trialJennifer M Kwon, Francina Munell, Laure Le Goff, et al.
Molecular Therapy. Methods & Clinical Development|December 10, 2024
Patient-specific responses to SMN2 splice-modifying treatments in spinal muscular atrophy fibroblastsIlaria Signoria, Maria M Zwartkruis, Lotte Geerlofs, et al.
Neurology|June 6, 2019
Natural course of scoliosis and lifetime risk of scoliosis surgery in spinal muscular atrophyCamiel A Wijngaarde, Rob C Brink, Floor A S de Kort, et al.
Neurology|July 31, 2012
Hexanucleotide repeat expansions in C9ORF72 in the spectrum of motor neuron diseasesWouter van Rheenen, Marka van Blitterswijk, Mark H B Huisman, et al.
Brain Communications|November 16, 2022
Population-based assessment of nusinersen efficacy in children with spinal muscular atrophy: a 3-year follow-up studyFéline E V Scheijmans, Inge Cuppen, Ruben P A van Eijk, et al.
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