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
Cindy Li, Ankit K. Desai, Punita Gupta, Katherine Dempsey, Vikas Bhambhani, Robert J. Hopkin, Can Fıçıcıoğlu, Pranoot Tanpaiboon, William J. Craigen, Amy S. Rosenberg, Priya S. Kishnani
Topics & Concepts
Enzyme replacement therapyMedicineRituximabInternal medicineNewborn screeningCreatine kinaseCohortDiseasePregnancyPediatricsAntibodyGastroenterologyImmunologyGeneticsBiologyLysosomal Storage Disorders ResearchGlycogen Storage Diseases and MyoclonusWhipple's Disease and Interleukins