Also known as: inherited leukoencephalopathies, hereditary white matter disorder
What is leukodystrophy?
Leukodystrophy refers to a large (more than 50), group of rare progressive diseases that affect the white matter of the brain (myelin). These conditions cause damage to the myelin sheath which slows or blocks messages between the brain and the rest of the body. There are a number of types including; Canavan disease, Krabbe disease, Niemann-Pick disease, and many others.
What causes leukodystrophy?
Genetic mutations appear to cause most leukodystrophies; they may be sporadic (not inherited from a parent) or hereditary disorders that can be passed along from parents to their children.
What are the symptoms of leukodystrophy?
Most symptoms usually appear during infancy or childhood but can start later. Early symptoms vary widely; common ones include loss of skills previously present, difficulty with walking/balance and mobility, cognitive decline, behavior and learning difficulties, bladder problems, seizures, and others.
Later symptoms include abnormal movements, changes in muscle tone, difficulties with eating and speaking, poor sight or hearing and declines in mental and physical development, depending on the nature and the area of the brain involved.
What are leukodystrophy care options?
Early diagnosis may enhance the chance for bone marrow or stem cell transplantation which when present damage, cannot be reversed; future damage caused by the disease however may be slowed. Other treatments are focused on managing the symptoms and supporting families; these include physical, occupational and speech therapy, learning support, nutrition management, medications for spasticity, seizures and other potential treatments.
This page was last updated on: 6/12/2018 10:08:50 AM
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Knowing how to swim saves lives. Swimming and water safety lessons are offered by a trained instructor for babies as young as 6 months to adolescents under 21 years old. Learn more.
Camp DMC is a summer program for children with special needs run by Nicklaus Children’s Dan Marino Outpatient Center. Please note: We will not offer Camp DMC during summer of 2019. Learn more.
This program is provided by a certified yoga instructor. It offers children and teens the following benefits: managing stress through breathing, self-awareness, healthy movement and meditation. Yoga also promotes strength, flexibility, coordination and body awareness. Learn more.
Yoga is a great way to get children active in a non- competitive environment. This one-day-a-week class is available for patients currently receiving therapy at one of our Nicklaus Children’s outpatient center locations, their siblings and children residing in our community. Learn more.