Lysosomal Storage Disease Center
The UCSF Lysosomal Storage Disease Center provides comprehensive care to patients with lysosomal storage disorders and their families. Our services include consultation, examination, testing, diagnosis, treatment and genetic counseling.
These inherited diseases are caused by problems with lysosomes, components of cells that contain enzymes (chemicals that break down various substances). When a particular enzyme is missing or inadequately supplied, these substances can build up to abnormal levels and cause symptoms.
There are many different lysosomal storage diseases. Treatment is currently available for Gaucher disease, Fabry disease and Hurler syndrome.
Because these diseases affect many different systems in the body, our team includes specialists in many disciplines. Our patients also have access to other experts at UCSF as well as Stanford.
Doctor referral required
Longitudinal Study of Urea Cycle Disorders
hyperammonemia, developmental disabilities, long-term renal and hepatic effects, and case-fatality associated with the various forms of UCD
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency
The incidence of adverse events (AEs), treatment-emergent adverse events (TEAEs), and serious adverse events (SAEs) for each dosing cohort, assessed by severity and relationship to study product.
Case Management and Social Work
Connect with a team that can help you find resources, solve problems and advocate for you during treatment at UCSF.
Mindfulness-Based Stress Reduction Class
This eight-week UCSF class teaches mindfulness practices, such as meditation and body awareness, that can reduce stress and improve your overall health.
We welcome feedback about your experience at UCSF Health. Find out how to contact us with comments, questions or concerns.
Spiritual Care Services
Chaplains representing many faiths are available around the clock to provide support, comfort and counsel to patients, families and caregivers. Learn more.
Plan your visit
What to Bring
- Photo I.D.
- Health insurance card
- Insurance authorization, if required
- Doctor's referral, if required
- Recent test results related to your condition
- List of your medications, including dosages, plus any you're allergic to
- List of questions you may have
- Device or paper for taking notes
If you live in another country and are coming to UCSF for medical
treatment, our patient liaisons can help coordinate all aspects of your visit.
UCSF offers language services at no charge for patients with limited
English, as well as for patients who are deaf or have trouble hearing.
Checking into the hospital? Find helpful information by clicking on the relevant “Your Hospital Stay” booklet: Mission Bay, Parnassus or Mount
Your Doctor Visit
See our top 10 tips for making your UCSF doctor’s appointment as stress-free and productive as possible.