Leukodystrophies: MedlinePlus

Leukodystrophies: MedlinePlus

 

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National Institutes of Health

Leukodystrophies

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The leukodystrophies are rare diseases that affect the cells of the brain. Specifically, the diseases affect the myelin sheath, the material that surrounds and protects nerve cells. Damage to this sheath slows down or blocks messages between the brain and the rest of the body. This leads to problems with

• Movement
• Speaking
• Vision
• Hearing
• Mental and physical development

Most of the leukodystrophies are genetic. They usually appear during infancy or childhood. They can be hard to detect early because children seem healthy at first. However, symptoms gradually get worse over time.
There are no cures for any of the leukodystrophies. Medicines, speech therapy and physical therapy might help with symptoms. Researchers are testing bone marrow transplantation as a treatment for some of the leukodystrophies.

NIH: National Institute of Neurological Disorders and Stroke

MEDICAL ENCYCLOPEDIA  

• Adrenoleukodystrophy
• Canavan disease
• Krabbe disease
• Metachromatic leukodystrophy

Related Topics

• Degenerative Nerve Diseases
• Brain and Nerves
• Genetics/Birth Defects
• Metabolic Problems

National Institutes of Health

• The primary NIH organization for research on Leukodystrophies is the National Institute of Neurological Disorders and Stroke

 

 

Start Here

• Leukodystrophy(National Institute of Neurological Disorders and Stroke) - Short Summary
• Also available in Spanish  

• Overviews

  • What Is Leukodystrophy(United Leukodystrophy Foundation)

• Diagnosis/Symptoms

  • Magnetic Resonance Imaging (MRI)(United Leukodystrophy Foundation)

• Treatment

  • Cerebral X-Linked Adrenoleukodystrophy (ALD) and Transplant(National Marrow Donor Program)
  • Krabbe Disease (Globoid-Cell Leukodystrophy)(National Marrow Donor Program)

• Specific Conditions

  • 4H Syndrome(United Leukodystrophy Foundation)
  • Adrenoleukodystrophy(National Institute of Neurological Disorders and Stroke) - Short Summary
  • Adrenomyeloneuropathy (AMN)(United Leukodystrophy Foundation)
  • Aicardi-Goutieres Syndrome Disorder(National Institute of Neurological Disorders and Stroke) - Short Summary
  • Alexander Disease(National Institute of Neurological Disorders and Stroke) - Short Summary
  • CADASIL(National Institute of Neurological Disorders and Stroke) - Short Summary
  • Canavan Disease(National Institute of Neurological Disorders and Stroke) - Short Summary
  • Cerebral Autosomal Dominant Arteriopathy with Subcortical Infacts and Leukoencephalopathy (CADASIL)(United Leukodystrophy Foundation)
  • Cerebrotendinous Xanthomatosis (CTX)(United Leukodystrophy Foundation)
  • Krabbe Disease(Mayo Foundation for Medical Education and Research)
  • Krabbe Disease(National Institute of Neurological Disorders and Stroke) - Short Summary
  • Megalencephalic Leukoencephalopathy with Subcortical Cysts (MLC)(United Leukodystrophy Foundation)
  • Metachromatic Leukodystrophy(National Institute of Neurological Disorders and Stroke) - Short Summary
  • Pelizaeus-Merzbacher Disease(National Institute of Neurological Disorders and Stroke) - Short Summary
  • Refsum Disease(National Institute of Neurological Disorders and Stroke) - Short Summary
  • Sjogren-Larsson Syndrome(United Leukodystrophy Foundation)
  • Types of Leukodystrophies(United Leukodystrophy Foundation)
  • Vanishing White Matter Disease(United Leukodystrophy Foundation)
  • Zellweger Syndrome(National Institute of Neurological Disorders and Stroke) - Short Summary
  • Return to top

• Clinical Trials

  • ClinicalTrials.gov: Adrenoleukodystrophy(National Institutes of Health)
  • ClinicalTrials.gov: Canavan Disease(National Institutes of Health)
  • ClinicalTrials.gov: Leukodystrophy, Globoid Cell(National Institutes of Health)
  • ClinicalTrials.gov: Leukodystrophy, Metachromatic(National Institutes of Health)
  • Return to top

• Genetics

  • Genetic Inheritance(United Leukodystrophy Foundation)
  • Genetics Home Reference: Aicardi-Goutieres syndrome(National Library of Medicine)
  • Genetics Home Reference: Alexander disease(National Library of Medicine)
  • Genetics Home Reference: Canavan disease(National Library of Medicine)
  • Genetics Home Reference: Hereditary diffuse leukoencephalopathy with spheroids(National Library of Medicine)
  • Genetics Home Reference: Hypomyelination and congenital cataract(National Library of Medicine)
  • Genetics Home Reference: Krabbe disease(National Library of Medicine)
  • Genetics Home Reference: Leukoencephalopathy with vanishing white matter(National Library of Medicine)
  • Genetics Home Reference: Megalencephalic leukoencephalopathy with subcortical cysts(National Library of Medicine)
  • Genetics Home Reference: Metachromatic leukodystrophy(National Library of Medicine)
  • Genetics Home Reference: Pelizaeus-Merzbacher disease(National Library of Medicine)
  • Genetics Home Reference: Pol III-related leukodystrophy(National Library of Medicine)
  • Genetics Home Reference: X-linked adrenoleukodystrophy(National Library of Medicine)
  • Genetics Home Reference: Zellweger spectrum(National Library of Medicine)
  • Return to top

• Journal Articles

  References and abstracts from MEDLINE/PubMed (National Library of Medicine)
  • Article: Medicine. Gene therapy that works.
  • Article: Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
  • Article: Clinical reasoning: a 47-year-old man with progressive gait disturbance and...
  • Leukodystrophies -- see more articles
  • Return to top

• Dictionaries/Glossaries

  • Leukodystrophy Glossary/Terms(United Leukodystrophy Foundation)
  • Return to top

• Directories

  • Genetic and Rare Diseases Information Center(Office of Rare Diseases)
  • Return to top

• Organizations

  • National Institute of Neurological Disorders and Stroke
  • Also available in Spanish
  • United Leukodystrophy Foundation