Mucopolysaccharidosis Type VII clinical trials at UCSF

2 research studies open to eligible people

Mucopolysaccharidosis Type VII is a rare genetic disorder that affects how the body breaks down sugars. UCSF is conducting a trial to test the safety of enzyme replacement therapy given to unborn babies. Another study at UCSF collects data from patients with lysosomal storage diseases to learn more about these conditions.

Showing trials for

  • PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

    open to eligible females ages 18-50

    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.

    San Francisco, California

  • Registry of Patients Diagnosed With Lysosomal Storage Diseases

    open to eligible people ages up to 64 years

    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.

    San Francisco, California

Our lead scientists for Mucopolysaccharidosis Type VII research studies include .

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