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2025
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2024
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2021
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Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naive patients with late-onset Pompe disease: A pooled analysis of clinical trials.
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S-EPMC11259910
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biostudies-literature
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A randomized EPIREMED protocol study on the long-term visuo spatial effects of very preterm children with a working memory deficit.
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S-EPMC8436542
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biostudies-literature
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Genome sequencing for the diagnosis of intellectual disability as a paradigm for rare diseases in the French healthcare setting: the prospective DEFIDIAG study.
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S-EPMC12495801
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biostudies-literature
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