PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

NCT04532047 · clinicaltrials.gov ↗

PHASE1

Phase

RECRUITING

Status

10

Enrollment

OTHER

Sponsor class

Conditions

MPS I
MPS II
MPS IVA
MPS VI
Mps VII
Gaucher Disease, Type 2
Gaucher Disease, Type 3
Pompe Disease Infantile-Onset
Wolman Disease

Interventions

DRUG: Aldurazyme (laronidase)

Sponsor

University of California, San Francisco

Collaborators

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