Girl, four, with 'childhood Alzheimer's' defies her death sentence to start ...

A four-year-old with a rare disease often referred to as 'childhood Alzheimer's' has defied the odds to start pre-school thanks to an experimental drug.

Marian McGlockin, four, was diagnosed with deadly Niemann-Pick disease type C (NPC) at just 18 months old.

The cruel condition normally kills children before they turn 10. It causes cholesterol to build-up in the neurons.

NPC leads to enlarged organs, lung damage, muscle stiffness, seizures, dementia and difficulty speaking. 

Marian's parents Sara, 36, and Paul, 35, say she wouldn't have made it to the age of four without the help of the drug known as VTS-270.

Marian McGlockin, four, was diagnosed with deadly Niemann-Pick disease type C (NPC) at just 18 months old

Marian McGlockin, four, was diagnosed with deadly Niemann-Pick disease type C (NPC) at just 18 months old

Her parents were warned she would lose her ability to talk, eat, walk, move, or even remember them

Her parents were warned she would lose her ability to talk, eat, walk, move, or even remember them

Marian's parents Sara, 36, and Paul, 35, say she wouldn't have made it to the age of four without the help of the drug known as VTS-270. Pictured together with Marian's older sister Emily, six

Marian's parents Sara, 36, and Paul, 35, say she wouldn't have made it to the age of four without the help of the drug known as VTS-270. Pictured together with Marian's older sister Emily, six

The youngster, from California, is now dancing (pictured wearing at her birthday party, playing and thriving at pre-school against the odds

The youngster, from California, is now dancing (pictured wearing at her birthday party, playing and thriving at pre-school against the odds

They took her to the doctors at the age of six months when they noticed a stunt in her growth.

Medics noticed she had an enlarged spleen - which eventually led to her diagnosis of NPC a year later. The disorder is incurable.

Her parents were warned she would lose her ability to talk, eat, walk, move, or even remember them. 

But the youngster, from California, is now dancing, playing and thriving at pre-school against the odds.

Her parents say she quickly showed vast improvements after taking part of a clinical trial of VTS-270, which is under review by Food and Drug Administration (FDA). 

The drug is injected into the spine of NPC sufferers and helps their bodies break down cholesterol clogging up their neurons. 

Mrs McGlockin, a mother-of-two, said: 'Marian has come on leaps and bounds over the past two years.

'She started learning to walk soon after starting to take the drug and even enjoys ballet classes now.

Marian's parents took her to the doctors at the age of six months (pictured as a baby) when they noticed a stunt in her growth

Marian's parents took her to the doctors at the age of six months (pictured as a baby) when they noticed a stunt in her growth

Medics noticed she had an enlarged spleen - which eventually led to her diagnosis a year later

Doctors told the family to prepare for the worst as the disease - dubbed 'childhood Alzheimer's' - normally kills in 10 to 15 years

Doctors told the family to prepare for the worst as the disease - dubbed 'childhood Alzheimer's' - normally kills in 10 to 15 years

Her parents say she showed vast improvements since taking part of a clinical trial of VTS-270, which is under review by Food and Drug Administration (FDA)

Her parents say she showed vast improvements since taking part of a clinical trial of VTS-270, which is under review by Food and Drug Administration (FDA)

The family say since starting on the medication, 'it's like it turned the lights on inside her and she really began to shine'

'In terms of her awareness and cognitive ability, she's where she should be for a four-year-old which is amazing as previously she was showing early signs of childhood dementia.

'We were terrified Marian would lose her ability to talk, eat, walk, move, and remember us.

WHAT IS NIEMANN-PICK DISEASE TYPE C (NPC)?

The rare disease sees patients unable to metabolise cholesterol and other lipids properly within their cells.

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Excessive amounts of cholesterol then accumulate within the liver and spleen, and excessive amounts of other lipids

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