Gene disorder with no cure

NIEMANN-Pick Type C is a rare inherited neurodegenerative disease with no known cure.

It is caused by an accumulation of lipids (fats) in the brain, liver and spleen.

Symptoms can include loss of memory and balance, lung and liver failure, tremors, delayed motor development, learning difficulties, dementia, swallowing problems and seizures.

Some victims are diagnosed in early childhood, whereas others may remain symptom-free until adulthood.

The earlier the onset of the disease, the shorter the life expectancy of the child.

Children inherit the disease when two copies of a mutated gene are passed on from both parents.