Wolman Disease clinical trials at UCSF
2 research studies open to eligible people
Wolman disease is a genetic disorder that prevents the breakdown of fats. UCSF is exploring prenatal enzyme replacement therapy through the PEARL trial. There is also a registry study collecting data internationally to better understand lysosomal storage diseases.
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 Tippi Mackenzie.
Last updated: