Wolman Disease clinical trials at UCSF

2 research studies open to eligible people

Wolman disease is a rare genetic disorder that affects the body's ability to break down fats. UCSF is studying how safe it is to treat unborn babies with enzyme replacement therapy. UCSF is also collecting data from patients worldwide to learn more about these types of diseases.

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 Wolman Disease research studies include .

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