High sustained antibody titers in patients with classic infantile Pompe disease following immunomodulation at start of enzyme replacement therapy
The Journal of Pediatrics Feb 16, 2018
Poelman E, et al. - Physicians designed this study to assess whether immunomodulation at the start of enzyme replacement therapy induced immune tolerance to recombinant human acid alpha-glucosidase (rhGAA) in patients with classic infantile Pompe disease. Only during B cell depletion, immunomodulation prevented the formation of rhGAA antibodies but failed to induce immune tolerance in 2/3 patients.
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