Publication | Open Access

Transforming the clinical outcome in CRIM-negative infantile Pompe disease identified via newborn screening: the benefits of early treatment with enzyme replacement therapy and immune tolerance induction

Maternal ImmunizationNewborn ScreeningAutoimmune DiseaseEnzyme Replacement TherapyImmunodeficienciesInherited Metabolic DiseasePathogenesisImmunologyPediatricsImmune Tolerance InductionAutoimmunityImmunologic DiseaseMedicineInborn Error Of Immunity