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European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|October 16, 2024
Developmental and epileptic encephalopathy 56 due to YWHAG variants: 12 new cases and review of the literatureMaria Eugenia Amato, Sol Balsells, Loreto Martorell, et al.
Molecular Therapy. Nucleic Acids|April 21, 2025
Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe diseaseRyan A Oliver, Meghan E Ahern, Perla G Castaneda, et al.
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