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Showing 1-2 of 2 trials for Pompe-disease-infantileonset
Recruiting

Registry of Patients Diagnosed With Lysosomal Storage Diseases

California · San Francisco, CA

This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.

Recruiting

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

California · San Francisco, CA

For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.