Pediatric Endocrinology Reviews
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Volume 17 Sup 1 - shop
Volume 17.2 - shop
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Therapy Development for the Lysosomal Storage Dise
Insights into the Pathogenesis and Treatment of Kr
CLN2 Disease (Classic Late Infantile Neuronal Cero
Types A and B Niemann-Pick Disease
The GM1 and GM2 Gangliosidoses: Natural History an
Juvenile NCL (CLN3 Disease): Emerging Disease-Modi
Prevention is the Best Therapy: The Geneticist’s A
Combination Therapies for Lysosomal Storage Diseas
Brain Targeting in MPS-IIIA