• Overview
• Inheritance
• Symptoms
  • Growth, Bones and Joints
  • Brain and Nerves
  • Physical Appearance
  • Ear, Nose, Throat and Chest
  • Heart
  • Liver, Spleen and Abdomen
  • Eyes
• Testing
• Treatment
• Resources
• Aldurazyme Update

Hurler Syndrome Resources

The International Patient Advocacy Association (IPAA)
The IPAA also provides resources and support to those affected by Fabry disease. The Association can help people with legal and insurance issues and can direct them to appropriate educational materials and patient meetings.

The Lysosomal Storage Disease Network (LSDN)
This group provides medical information on lysosomal storage diseases, including Fabry disease. The LSDN also helps to link academic medical centers that treat patients with these conditions, so that health care professionals may share their expertise with one another. The LSDN is administered by Transkaryotic Therapies, manufacturers of Replagal.

MPS I Registry
This registry was established in April 2003 and is sponsored by Genzyme Corporation and BioMarin Pharmaceuticals. The registry collects and analyzes clinical information on people with MPS I. The goal is to increase the medical understanding of MPS I and its treatment. If you are a patient at the Center, and you would like to be enrolled, contact Cindy Morgan, MS.

The National MPS Society
The Society provides support services, including conferences, written information, a members' directory, and a newsletter, to people with MPS diseases (including Hurler syndrome) and their families. The Society also funds fellowships and research studies related to MPS diseases.

The Canadian MPS Society
The Society provides support services, including conferences, written information, a quarterly newsletter, and an on-line message board, to people with MPS diseases (including Hurler syndrome) and their families. The Society also funds MPS-related research.

The National Tay-Sachs and Allied Diseases Association (NTSAD)
NTSAD administers a program called the Parent Peer Group, a support group of about 400 families with Hurler syndrome and related diseases. The NTSAD also maintains a resource list of health care workers, therapists, and other professionals familiar with these diseases.

The National Organization for Rare Disorders (NORD)
NORD is dedicated to the identification, treatment, and cure of all rare diseases, which affect fewer than 200,000 Americans. NORD maintains a medical information database for these disorders, as well as a support group database. The organization also provides grants for researchers, a networking program for patients, and many other services.

UCSF's Pediatrics Bone Marrow Transplant Program
A team of specialists performs bone marrow transplants for pediatric patients at UCSF. Patients with Hurler syndrome who are candidates for bone marrow transplantation are referred to the Program for discussion and transplant-related care.