In this review:
Diffusion tensor imaging in children with PD
Enzyme replacement therapy effects on cardiac function and structure
In utero enzyme replacement therapy for PD
Early clinical phenotype of LOPD
Immunogenicity of cipaglucosidase-α/miglustat in LOPD
Avalglucosidase-α and HRQoL in LOPD
Liver-directed and systemic AAV gene transfer approaches for PD therapy
Parents supportive care after newborn screening LOPD diagnosis
Transcriptomic profiling of skeletal muscle in LOPD
Tolerance of genetic reduction of muscle glycogen
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