Journal of pediatric endocrinology & metabolism : JPEM
Successful desensitization to alglucosidase alfa in a very young infant with Pompe disease.
Funda Aytekin Güvenir, Gökhan Yörüsün, Zeynep Şengül Emeksiz, Oya Kıreker Köylü, Aynur Küçükçongar Yavaş, Emine Dibek Mısırlıoğlu
Published: 202610.1515/jpem-2025-0599
Abstract
OBJECTIVES: Enzyme replacement therapy (ERT) with alglucosidase alfa markedly improves outcomes in infantile-onset Pompe disease, yet hypersensitivity reactions may interrupt treatment and require desensitization. We report a very young infant in who…
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