Severe CNS involvement in a subset of long-term treated children with infantile-onset Pompe disease
Daniel Kenney‐Jung, Aditi Korlimarla, Gail A. Spiridigliozzi, Walter F. Wiggins, Michael D. Malinzak, Gretchen Nichting, Seung‐Hye Jung, Angela Sun, Raymond Wang, Aisha Al Shamsi, Chanika Phornphutkul, James W. M. Owens, James M. Provenzale, Priya S. Kishnani
Topics & Concepts
HyperintensityMedicineEncephalopathyPediatricsEnzyme replacement therapyMagnetic resonance imagingHyperreflexiaDiseaseInternal medicineSurgeryRadiologyLysosomal Storage Disorders ResearchGlycogen Storage Diseases and MyoclonusTrypanosoma species research and implications