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PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) — Stella

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PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Phase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.

Trial locations(1 site)

United States

University of California, San Francisco, California

Key details

Phase

Early testing

Sponsor

University of California, San Francisco

Collaborators

Duke University

Enrollment target

~10 participants

Started

July 2021

Primary completion

July 2031

Age range

18 Years – 50 Years

Sex

Female only

Last updated March 2026

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