Virtual Information Session
Thursday, April 27, 2023, 8:00-9:30 AM PDT check your time zone
Register: https://tinyurl.com/IUERT
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
