Leukodystrophies: diseases that particularly affect children

These are genetic diseases that destroy the myelin (nerve sheath) of the nervous system. When this sheath is damaged, the current no longer passes and the nerve messages are interrupted, which rapidly leads to the deterioration of all functions (sight, hearing, motor skills, memory, etc.) and very often to death.

The diseases:

• ALD/AMN
• Undefined leukodystrophies
• Canavan disease
• Krabbe disease
• Refsum disease
• Alexander disease
• MLC disease
• MLD
• PMD and other hypomyelinating leukodystrophies
• Aicardi-Goutières syndrome
• CACH Syndrome
• LTBL

Some key figures

• 3 to 6 children per week are born with leukodystrophy in France (20 to 40 in Europe).
• €48.8 million invested through 571 leukodystrophy research programs.
• €15.7 million devoted to support families.