Infantile-onset Pompe disease complicated by sickle cell anemia: Case report and management considerations
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...
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Frontiers Media S.A.,
2022-09-01T00:00:00Z.
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