Lysosomal Storage Program

Lysosomal Storage Program Overview

LIke ThisLIke ThisLIke ThisLIke ThisLIke This

Boston Children's Hospital's Lysosomal Storage Program is a multidisciplinary clinic that diagnoses and treats children and adults with known or suspected lysosomal diseases. These inherited diseases occur when one or more of a child's enzymes is either missing or not working correctly. Without these enzymes, harmful substances can build up in a child's body and cause developmental delays and mental impairment. Examples of lysosomal storage disorders are Gaucher disease and Tay Sachs disease.

Boston Children's established the Lysosomal Center for Diagnosis and Treatment, the first treatment center to focus specifically on the treatment of these rare and often fatal diseases, in 2003. The center was established to bring new treatment options for these rare disorders to patients from around the world.

A multidisciplinary approach

Our center at Boston Children's brings together specialists from different departments, including Genetics, Metabolism, Cardiology and Neurology, to treat your child's specific symptoms and needs.

Support groups and resources

In addition to diagnosing and treating the disease, we maintain close ties with family support groups that provide families facing similar challenges with additional support and helpful educational information.

Request an Appointment

If this is a medical emergency, please dial 9-1-1. This form should not be used in an emergency.

Patient Information
Date of Birth:
Contact Information
Appointment Details
Send RequestIf you do not see the specialty you are looking for, please call us at: 617-355-6000.International visitors should call International Health Services at +1-617-355-5209.
Please complete all required fields

This department is currently not accepting appointment requests online. Please call us at: 617-355-6000. International +1-617-355-6000.

This department is currently not accepting appointment requests online. Please call us at: 617-355-6000. International +1-617-355-6000.

Thank you.

Your request has been successfully submitted

You will be contacted within 1 business day.

If you have questions or would like more information, please call:

617-355-6000 +1-617-355-6000
close
Find a Doctor
Search by Clinician's Last Name or Specialty:
Select by Location:
Search by First Letter of Clinician's Last Name: *ABCDEFGHIJKLMNOPQRSTUVWXYZ
More optionsSearch
Condition & Treatments
Search for a Condition or Treatment:
Show Items Starting With: *ABCDEFGHIJKLMNOPQRSTUVWXYZ
View allSearch
Visitor Information
The future of pediatrics will be forged by thinking differently, breaking paradigms and joining together in a shared vision of tackling the toughest challenges before us.”
- Sandra L. Fenwick, President and CEO
Close