Hurler syndrome medical therapy

Jump to navigation Jump to search

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]

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 treated with 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-Scheie syndrome that does not cause mental retardation.

References