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    Desensitization of two young patients with infantile-onset Pompe disease and severe reactions to alglucosidase alfa

    Pompe disease is a metabolic myopathy, due to deficiency of alpha glucosidase, with a wide clinical spectrum. Enzyme replacement therapy is the only available treatment to improve morbidity and mortality, espe...

    V. Gragnaniello, S. Fecarotta, A. Pecoraro, A. Tarallo, A. Catzola… in Neurological Sciences (2019)