Who is the oldest living person with Batten disease?

There isn't one definitive "oldest living person" with Batten disease due to the rarity and varied forms of the condition, but individuals like Malachi Castilotte (as of early 2025) have been noted as among the oldest living children receiving treatment in the U.S. for specific types (CLN2), while figures like the late Nathan Milto previously held records, showing some patients can live into their 20s or 30s with supportive care and newer treatments.

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What is the end stage of Batten disease?

End-stage Batten disease is when your child needs around-the-clock care due to severe symptom progression. At this stage, your child may not be able to move or get out of bed.

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How many people in the world have Batten disease?

Some children die in early childhood, while others may be able to live into their teens or twenties. Worldwide, roughly 14,000 children are known to have Batten disease. In the U.S., it affects an estimated 2 to 4 out of every 100,000 children. There is currently no cure.

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What medication is used for Batten disease?

In 2017, cerliponase alfa (Brineura), a tripeptidyl peptidase enzyme replacement therapy, became the first globally approved treatment for CLN2 Batten disease.

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Has anyone ever survived Batten disease?

Tragically, the disease is fatal. There are some treatments which can help with some of the symptoms of juvenile Batten disease, such as seizures. But there is no treatment that can slow the progression of the disease, which is caused by problems with a specific gene and is an inherited neurodegenerative condition.

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Living with Batten Disease - Three journeys

43 related questions found

Is Batten's disease always fatal?

Over time, affected children suffer mental impairment, worsening seizures, and progressive loss of sight and motor skills. Children become totally disabled and eventually die. Batten disease is not contagious nor, at this time, preventable. To date it has always been fatal.

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What is the longest anyone has lived with trisomy 18?

Megan Hayes, oldest known individual in the US (2nd oldest in the world) with Full Trisomy 18 has recently turned 40 years old.

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Does Batten disease run in families?

Batten disease is typically inherited in an autosomal recessive manner. This means that two copies of a mutated gene are needed to cause the disease. In most cases, one copy is inherited from each parent. Parents, who have only one copy of the mutated gene each, are unaffected.

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What age does Batten disease start?

Batten disease is a fatal, inherited disorder of the nervous system that typically begins in childhood. Early symptoms of this disorder usually appear between the ages of 5 and 10 years, when parents or physicians may notice a previously normal child has begun to develop vision problems or seizures.

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Can gene therapy cure neurodegenerative diseases?

Gene therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of neurodegenerative disorders, including Alzheimer's disease (AD), Parkinson's disease (PD) and Huntington's disease (HD). Some early clinical trials have failed to achieve satisfactory therapeutic effects.

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What is the deadliest genetic disease?

Cystic fibrosis (CF) is the most common fatal genetic disease in the United States today. It causes the body to produce a thick, sticky mucus that clogs the lungs, leading to infection, and blocks the pancreas, stopping digestive enzymes from reaching the intestines where they are required to digest food.

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How many people in Australia have Battens disease?

It's incredibly rare and according to the Batten Disease Support and Research Association about 35 children have some form of Batten disease in Australia — eight of which have CLN2.

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What is the end stage of neurodegenerative disease?

The end of life or palliative phase in neurodegenerative illness is defined as the last 6 to 12 months (though unpredictable declines can occur). The last days of life are typically the final 2–3 days but may be several weeks.

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Does Batten disease hurt?

Batten disease is a rare neurodegenerative and fatal inherited disorder of childhood. Vision loss, seizures, hallucinations, and mental and motor function impairment emerge after seemingly typical development. Little is known about the pain experience of individuals with Batten disease.

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What is the life expectancy of a person with vascular dementia?

Life expectancy with vascular dementia varies widely but averages around 5 years after diagnosis, often less than Alzheimer's, because it shares risk factors with heart attack/stroke, and death is often due to these conditions rather than the dementia itself, though many die from their dementia. Prognosis depends heavily on overall health, age, sex (men often have shorter survival), comorbidities, and stroke/heart attack history, with progression marked by plateaus and sudden declines.
 

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Can Batten disease be misdiagnosed?

Because Batten disease is rare and many pediatricians may not have encountered it, it can be difficult to get an accurate diagnosis. Some children are incorrectly diagnosed with seizure disorders or autism. Currently, most diagnoses of Batten disease are made by genetic testing.

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What triggers neurodegeneration?

Common pathogenic mechanisms underlying many NDDs include: Abnormal protein dynamics with misfolding, defective degradation, proteasomal dysfunction and aggregation; often with actions and mutations of molecular chaperones; Oxidative stress (OS) and formation of free radicals/reactive oxygen species (ROS);

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What's the youngest age you can get Parkinson's?

While Parkinson's disease is usually age-related, it can happen in adults as young as 20 (though this is extremely rare, and often people have a parent, full sibling or child with the same condition).

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Can Batten disease be detected in pregnancy?

We report on the use of direct gene analysis in the prenatal diagnosis of this disease. Methods and findings A Finnish woman with a son with Batten's disease came for genetic counselling for her current pregnancy. Electron microscopy of a chorionic villus sample gave suggestive findings.

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Which parent do you inherit the most from?

Genetically, a person actually carries more of his/her mother's genes than his/her father's. The reason is little organelles that live within cells, the? mitochondria, which are only received from a mother. Mitochondria is the powerhouse of the cell and is inherited from the mother.

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What is 90% of autism caused by?

Around 90% of autism cases are attributed to genetic factors, meaning autism is highly heritable, with many different genes contributing, rather than a single cause, often interacting with environmental influences during early brain development, though specific environmental factors don't cause it but can increase risk. Twin studies show strong genetic links, with concordance rates between 60-90% in identical twins, and research points to complex interactions of many genes and prenatal/perinatal factors. 

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Why do I only share 47% DNA with my dad?

It is not uncommon for Ancestry Composition Inheritance to report that a son or daughter inherited slightly more or less than 50% from each parent. This is because Ancestry Composition relies on the autosomes (chromosomes 1–22) and the X chromosome(s) to calculate Inheritance.

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Can you have a baby with trisomy 18 twice?

The chance of having another baby with trisomy 18 or 13 is no more than 1 percent, although the risk increases slightly as the mother ages.

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