Clinical Trial Update: PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) [Phase 1]

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Summary

Phase 1 prenatal enzyme replacement therapy trial PEARL is currently recruiting for multiple lysosomal storage disorders including MPS I, MPS II, MPS IVA, MPS V…

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Diseases / indications

• Gaucher Disease Type
• Mucopolysaccharidosis Type
• Mucopolysaccharidosis Type Ii
• Mucopolysaccharidosis Type Iv
• Mucopolysaccharidosis Type Vi
• Mucopolysaccharidosis Type Vii
• Pompe Disease

Drugs mentioned

• PEARL

Snippet

Sponsor: University of California, San Francisco | Conditions: MPS I, MPS II | Phase 1 | Status: RECRUITING