Infantile-onset Pompe disease complicated by sickle cell anemia: Case report and management considerations

• Main Authors: Rodrigo Tzovenos Starosta (Author), Ying-Chen Claire Hou (Author), Katelyn Leestma (Author), Prapti Singh (Author), Luke Viehl (Author), Linda Manwaring (Author), Jorge Luis Granadillo (Author), Molly C. Schroeder (Author), Jamie N. Colombo (Author), Halana Whitehead (Author), Patricia Irene Dickson (Author), Monica L. Hulbert (Author), Hoanh Thi Nguyen (Author)
• Format: Book
• Published: Frontiers Media S.A., 2022-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Infantile-onset Pompe disease (IOPD) is a rare, severe disorder of lysosomal storage of glycogen that leads to progressive cardiac and skeletal myopathy. IOPD is a fatal disease in childhood unless treated with enzyme replacement therapy (ERT) from an early age. Sickle cell anemia (SCA) is a relativ...