Wed. Nov 6th, 2024
alert-–-virginia-girl,-8,-is-suffering-‘childhood-dementia’-as-her-body-slowly-shuts-down-from-rare-terminal-neurological-illness-that’s-been-crippling-her-body-since-she-was-just-three-months-oldAlert – Virginia girl, 8, is suffering ‘childhood dementia’ as her body slowly shuts down from rare terminal neurological illness that’s been crippling her body since she was just three months old

The family of a young girl from Virgina who suffers from ‘childhood dementia’ has watched as her body slowly shuts down from a rare, terminal neurological illness. 

Eight-year-old Abby Alvey was diagnosed with Niemann Pick Type C – an ultra-rare genetic disease – when she was four after an extensive diagnostic journey.  

The rare disease causes physical and neurological symptoms, including dementia at a young age. 

Abby’s father Garland Alvey told WTVR the disease is ‘just the most disgusting, horrible thing you could ever imagine.’ 

He said when she was just a couple months old, she began to bruise easily, and her liver and spleen were swelling- telltale signs of the incurable disease. 

Abby Alvey was diagnosed with Niemann Pick Type C- an ultra-rare genetic disease- when she was four years old. She started to show symptoms when she was just a couple months old

Abby Alvey was diagnosed with Niemann Pick Type C- an ultra-rare genetic disease- when she was four years old. She started to show symptoms when she was just a couple months old 

Abby pictured with her older sister and her father Garland who have both supported her throughout her journey

Abby pictured with her older sister and her father Garland who have both supported her throughout her journey 

Abby goes through extensive testing and screenings for the rare disease and is set to start biweekly spinal infusions to ease her symptoms

Abby goes through extensive testing and screenings for the rare disease and is set to start biweekly spinal infusions to ease her symptoms 

At the age of three Abby started to lose her balance and her speech changed. 

The life expectancy for people with Niemann Pick Type C can vary, while some only make it to childhood and other make it to adulthood. 

It is unclear how much life Abby has left to live, but her dad said that she might not be able to make it to high school.

‘No matter what we do, we can’t stop that progression. You go through a very dark phase of ‘how are we going to handle this’,’ Garland said.     

With Abby’s fait hanging over the family, they have decided to try and live every day to the fullest and cherish every moment they have with her. 

She goes to school, participates in dance classes, enjoys arts and crafts and loves to sing along to Disney songs. 

Her parents created a superhero inspired after their brave daughter named ‘Abby Strong’ as a way to remind her of her and others strength dealing with the disease.

Alvey said: ‘She’s Abby Strong, and she is fighting for all of her NPC buddies. Go and do as much as we possibly can and live a lifetime within a short amount of time.’ 

A Facebook page has also been made in honor of Abby and her journey and is called AbbyStrong Fights NPC. 

Her parents created a superhero inspired after their brave daughter named 'Abby Strong' as a way to remind her of her and others strength dealing with the disease

Her parents created a superhero inspired after their brave daughter named ‘Abby Strong’ as a way to remind her of her and others strength dealing with the disease

Garland said that when Abby was just a few months old, they noticed that she would bruise very easily, and her liver and spleen were swelling

Garland said that when Abby was just a few months old, they noticed that she would bruise very easily, and her liver and spleen were swelling 

Her father has also set up a gofundme page for Abby which is meant for others to donate toward the costs of her frequent medical treatments, devices and therapies to help her ‘live a longer fuller life’. 

The donation page revealed that Abby has multiple therapies throughout the week, and she will soon start biweekly spinal infusions.

The infusions will administer a drug into her body that will help with the buildup of ‘toxic waste from her cells,’ dad said on the page. 

Niemann Pick Type C or NPC is caused by an ability of the body to transport cholesterol and other fatty substances like lipids inside of cells that the body can’t properly break down. 

The excessive fatty cells then expand on to other parts of the body’s tissue, including brain tissue, which leads patients to suffer from dementia. 

These dementia symptoms usually consist of poor judgement skills and issues with short and long-term memory and learning.   

Other symptoms that people with NPS experience are an enlarged liver and spleen, difficulty coordinating movement, abnormal eye movements, severe liver disease and difficulty with speech. 

Though symptoms of NPC tend to occur during childhood, they can truly begin at any age, and approximately 500 cases of the rare metabolic disease have been diagnosed worldwide. 

Abby’s family also has hopes of taking her to Disney World soon as she loves to sing along to songs and express herself. 

Abby goes to school, participates in dance lessons,cv

Abby goes to school, participates in dance lessons, enjoys arts and crafts and loves to sing along to Disney songs 

Her father has also set up a gofundme page for Abby which is meant for others to donate toward the costs of her frequent medical treatments, devices and therapies to help her 'live a longer fuller life'

Her father has also set up a gofundme page for Abby which is meant for others to donate toward the costs of her frequent medical treatments, devices and therapies to help her ‘live a longer fuller life’

They also hope that her story helps encourage others to peruse newborn generic testing and screenings for rare diseases.  

Many people with rare diseases go through strenuous amounts of testing and often get misdiagnosed before they reach their diagnosis. 

As of 2023, there are an estimated 7,000 known rare diseases that affect more than 300 people worldwide, according to Sanofi. Rare Disease Day is celebrated annually on the last day of February.  

‘She deserves a chance just like everyone else to do the things we all do and sometimes take for granted. We are not going to just hide and give up,’ Alvey said.

What is Niemann-Pick disease type C?

Niemann-Pick disease type C, also known as NPC, is an incredibly rare, inherited disease which causes damage to the nervous system over time.

An estimated one in 120,000 people are born with the condition.

The life expectancy of people with NPC varies. Some people die in childhood while others survive into adulthood.

Children who show symptoms after entering school may live into their mid-to-late teens, with a few surviving into their 20s.

A 2019 study found that the median life expectancy of sufferers was 13.

NPC is caused by an ability of the body to transport cholesterol and other fatty substances (like lipids) inside of cells.

Because the body is unable to process these fats and properly break them down, they build up in various tissues of the body, including brain tissue.

Symptoms of NPC tend to occur in childhood, but they can begin at any age. They include: enlarged liver and spleen, difficulty coordinating movement, abnormal eye movements, severe liver disease and difficulty with speech.

People with NPC are more likely to develop symptoms of dementia such as poor judgement, problems with short-term memory and learning.

Currently, there is no cure for NPC. There is a drug called Miglustat which stops the body from producing excess lipids which helps to slow progression of the disease.

NPC is caused by a mutation in either the NPC1 or NPC2 genes, which give instructions for the production of special proteins in lysosomes that are responsible for the movement of cholesterol and other fats.

Lysosomes are sacs filled with enzymes that can break down or digest materials inside cells, such as cholesterol and lipids.

The condition is inherited when a child receives a defective copy of the NPC1 or NPC2 gene from each of their parents.

NPC is one of about 50 diseases classified as lysosomal storage disorders (LSD), where a genetic variation disrupts the normal activity of lysosomes in human cells.

 

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