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Lysosomal Storage Disease Center

The Lysosomal Storage Disease Center provides comprehensive care to children, teens and adults and their families living with lysosomal disease, which includes about 50 rare inherited metabolic disorders. Our center offers consultations, evaluation, testing, and diagnosis and treatment of these disorders, with a special focus on Gaucher disease, Fabry disease and Hunter syndrome.

Lysosomal storage disorders are caused by defects of the lysosomes, parts of a cell that break down fats and carbohydrates so the body can recycle them. Without the work of digestive enzymes that lyosomes contain, toxic substances build up and can damage bodily tissues and organs over time. Symptoms of lysosomal storage disease vary in severity, depending on the particular missing enzymes and other factors such as age of diagnosis, but can include developmental delays, movement disorders, seizures, dementia, deafness, blindness and lung and heart problems.

Because these diseases affect many different systems in the body, our team includes specialists in a variety of disciplines, and we work closely with other UCSF experts as needed.

Our locations (1)

    Awards & recognition

    • Ranked among the nation's best in 11 specialties

    Plan your visit

    Preparing for your appointment

    What to Bring

    • Photo I.D.
    • Health insurance card
    • Insurance authorization, if required
    • Doctor's referral, if required
    • Recent test results related to your child's condition
    • List of medications, including dosages, plus any your child is allergic to
    • List of questions you may have
    • Device or paper for taking notes

    For visitors

    Before you head to the hospital, check out our guidelines for visitors and caregivers.

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    Your Stay

    We offer valuable information to help you plan for your child's upcoming hospital stay. Find out about food services, family accommodations and more.

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    Your Child's Care Team

    Our doctors, nurses, child life specialists and other providers have special training to care for children. Meet your child's care team.

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    Interpreter Services & Communication Assistance

    Interpreter services in many languages and TDDs are available for families that need help communicating with care teams. Here's how to access them.

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    Support services

    Child Life Services

    Activity rooms, pet therapy and more to help kids feel at home.

    Child Life

    Certified child life specialists ease the stress and anxiety of childhood illness through therapeutic play, schooling and family-focused support.

    Family Amenities

    Family-friendly amenities help you relax and take care of yourself while staying close to your child. We offer lounges, kitchens, showers, breastfeeding rooms and more.

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    Social Services

    Social workers ease the effect of illness, injury and hospitalization on your family with counseling and assistance to help you navigate the challenges.

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    Activity Rooms

    Activity rooms offer a relaxed environment for children of all ages. Features include a playroom, digital arts studio and teen lounge. Find out more.

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    Art Therapy

    Art therapy helps kids and families express themselves, making it easier to cope with pediatric health issues. Group and bedside services are available.

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    Music Therapy Program

    Board-certified music therapists help kids cope with illness and the feelings that come with it. Find out about group activities and bedside therapy.

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    School Program

    Our schoolroom services and individual learning programs help children hospitalized at UCSF continue their education and get ready to return to school.

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    School Re-Entry Program

    We help kids return to school after hospitalization by educating staff and students about their needs and recommending needed accommodations. Read more.

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    Individual Playtime & Instruction

    If your child can't be around other kids for medical reasons, you can sign up for private sessions in our activity and school rooms. Find out how.

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    Interpreter Services & Communication Assistance

    Interpreter services in many languages and TDDs are available for families that need help communicating with care teams. Here's how to access them.

    Family Resource Center

    We help families cope with the challenges of childhood illness and hospitalization in a relaxing environment where parents can get a much-needed break.

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    Parent Support Program

    Weekly activities, including knitting, scrapbooking, chair massage and more, help family caregivers get a much-needed break. Find out more.

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    Patient Relations

    Patient relations reps and nursing supervisors are here to answer questions and address concerns. Learn about your rights, how to reach us and more.

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    Spiritual Care

    Chaplains help UCSF patients, families and staff cope with the spiritual and emotional challenges of childhood illness. Learn more.

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    International Services

    If you're coming to UCSF from outside the U.S., we coordinate your child's appointments and provide other helpful services. Here's how to get started.

    Our research initiatives

    • ucsf-institute-for-human-genetics-research-2x

      UCSF Institute for Human Genetics Research

    Saving future lives

    500+ UCSF investigators are researching cures for hundreds of childhood and adult diseases.

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