Early ERT Key in Severe Classic Infantile Pompe

Early ERT Key in Severe Classic Infantile Pompe

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Early ERT Key in Severe Classic Infantile Pompe

Early treatment with enzyme replacement therapy (ERT) and immune tolerance induction (ITI) greatly improved clinical outcomes for infants with the severe, frequently ERT-nonresponsive, CRIM-negative infantile Pompe disease (IPD), a study has found. “Our data suggest that the first few weeks after birth may be a critical period in newborns with CRIM-negative IPD, during which clinical symptoms may not yet be fully apparent, but timely treatment initiation can result in drastically improved clinical outcomes,” the investigators wrote. The…

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