genetic
Metachromatic leukodystrophy
Metachromatic leukodystrophy (MLD) is a storage disease which is commonly listed in the family of leukodystrophies as well as among the sphingolipidoses as it affects the of sphingolipids. Leukodystrophies affect the growth and/or development of myelin, the fatty covering that acts as an insulator around nerve fibers throughout the central and nervous systems. MLD involves cerebroside sulfate accumulation. Metachromatic leukodystrophy, like most deficiencies, has an inheritance pattern.
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Symptoms — what it feels like
- ·Progressive neurologic decline
Causes — why it happens
- · storage disease
How it's found
- · based and
Treatment
- ·HSCT (pre-), Gene therapy (late infantile), Palliative
Complications
- ·, , loss of motor skills
Outlook
- ·fatal
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