For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.
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Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
Timeframe: 6 years
Number of participants to receive the full initial, weight-based dose of enzyme replacement therapy through the fetal umbilical vein, and subsequent doses throughout the pregnancy.
Timeframe: 6 years
Number of participants with the presence and levels of glycosaminoglycans (GAGs) in urine.
Timeframe: 6 years
The number of participants with improvement or resolution of hydrops (if present).
Timeframe: 6 years