In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

key information

study id #: NCT04532047

condition: MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease

status: Recruiting


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.

intervention: Aldurazyme (laronidase)


last updated: February 26, 2022