To evaluate whether immunomodulation at the start of enzyme replacement therapy (ERT) prevents formation of anti-recombinant human acid alpha-glucosidase (rhGAA) antibodies in classic infantile Pompe patients. Patients were treated with 4 weekly doses of Rituximab, weekly administration of Methotrexate, monthly intravenous immunoglobulins and alglucosidase alfa 40 mg/kg/week. Antibody titers were measured using ELISA and immunoprecipitation, also their neutralizing effects were determined. Clinical efficacy was evaluated by (ventilator-free) survival, reduction in left ventricular mass index and improvement of motor function.

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