Join us for this virtual information session regarding Prenatal Enzyme Replacement Therapy for Lysosomal Storage Diseases. We will present preliminary results from a phase 1 clinical trial (NCT04532047) to treat eight lysosomal storage diseases prenatally by infusing the fetus with weight-adjusted doses of recombinant enzymes currently approved for use in pediatric patients.

Conditions include MPS 1, 2, 4a, 6, 7; Gaucher Disease Type 2 and 3; Infantile-onset Pompe Disease; and Wolman Disease.

In this session, Dr. Tippi MacKenzie, Professor of Surgery at the University of California, San Francisco, and colleagues from UCSF will discuss the following:

· Preclinical studies for in utero enzyme replacement therapy (IUERT)
· Rationale and Investigational New Drug approval for IUERT
· Inclusion/exclusion criteria and the role of the Enrollment Advisory Board
· Clinical protocol and care of patients through the trial
· Case review of the first patient treated using the IUERT clinical protocol (Infantile-onset Pompe Disease)
· Preliminary information on two additional patients treated with IUERT
· Considerations for in utero infusions of ERT via the umbilical vein
· Considerations for postnatal care, including clinical trials of gene therapies

Register and join us on April 27 to learn about this innovative approach to treat lysosomal storage diseases before birth.

Please share this event with colleagues and patients who might be interested in learning more.  All are welcome to attend.