Hurler syndrome medical therapy

Hurler Syndrome Microchapters
Home
Patient Information
Overview
Historical Perspective
Classification
Pathophysiology
Causes
Differentiating Hurler Syndrome from other Diseases
Epidemiology and Demographics
Risk Factors
Screening
Natural History, Complications and Prognosis
Diagnosis
History and Symptoms
Physical Examination
Laboratory Findings
Electrocardiogram
X Ray
CT
MRI
Echocardiography or Ultrasound
Other Imaging Findings
Other Diagnostic Studies
Treatment
Medical Therapy
Surgery
Primary Prevention
Secondary Prevention
Cost-Effectiveness of Therapy
Future or Investigational Therapies
Case Studies
Case #1
Hurler syndrome medical therapy On the Web
Most recent articles
Most cited articles
Review articles
CME Programs
Powerpoint slides
Images
American Roentgen Ray Society Images of Hurler syndrome medical therapy All Images X-rays Echo & Ultrasound CT Images MRI
Ongoing Trials at Clinical Trials.gov
US National Guidelines Clearinghouse
NICE Guidance
FDA on Hurler syndrome medical therapy
CDC on Hurler syndrome medical therapy
Hurler syndrome medical therapy in the news
Blogs on Hurler syndrome medical therapy
Directions to Hospitals Treating Hurler syndrome
Risk calculators and risk factors for Hurler syndrome medical therapy

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Aditya Govindavarjhulla, M.B.B.S. [2]

Please help WikiDoc by adding more content here. It's easy! Click here to learn about editing.

Overview

Treatment of Hurler syndrome involves supplementing the body with the deficient enzyme so that it can break down the GAG's. Enzyme replacement therapy has proven useful in reducing non-neurological symptoms and pain.

Medical Therapy

Enzyme Replacement Therapy

• Patient is given the drug Laronidase, or Aldurazyme.
• This is is a polymorphic variant of Alpha-L-iduronidase (lysosomal enzyme), produced by recombinant DNA technology.
• This enzyme is required for the hydrolysis of terminal Alpha-L-iduronic acid residues of heparan and dermatan sulfate.
• Enzyme replacement therapy is useful in dealing with problems of growth, breathing, heart and joints.
• It is not proven effective in cases of neurological symptoms or mental development problems.
• It is a good option for children with Scheie syndrome or Hurler's/Scheie syndrome that does not cause mental retardation.