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Prenatal Enzyme Replacement Therapy for Lysosomal Storage Diseases

Virtual Information Session

Thursday, April 27, 2023, 8:00-9:30 AM PDT check your time zone


Join us to learn about the phase 1 clinical trial (NCT04532047) at UCSF to treat lysosomal storage diseases prenatally by infusing the fetus with weight-adjusted doses of recombinant enzymes currently approved for use in pediatric patients. 

Conditions included in the study: MPS 1, 2, 4a, 6, 7; Gaucher Disease Type 2 and 3; Infantile-onset Pompe Disease; and Wolman Disease. 

This session will cover:

  • Rationale for in utero enzyme replacement therapy (IUERT) 
  • Preclinical studies leading to Investigational New Drug approval
  • Case review of the first patient treated with the IUERT clinical protocol (Infantile-onset Pompe Disease) 
  • Preliminary information on two additional patients treated with IUERT 
  • Inclusion/exclusion criteria and clinical protocol
  • Considerations for in utero infusions of ERT via the umbilical vein 
  • Postnatal care, including clinical trials of gene therapies

All are welcome!