Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early‐Onset Lysosomal Storage Diseases

Abstract excerpt

The expansion of prenatal genetic screening and diagnosis warrants the evaluation of approved postnatal therapies that may be safely and feasibly translated to prenatal administration to a fetus affected by monogenic disease. For lysosomal storage diseases (LSDs), enzyme replacement therapy (ERT) often represents the main therapeutic approach. In utero enzyme replacement therapy (IUERT) has several potential...

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