Alternating hemiplegia of childhood / Disease or Syndrome

Understanding alternating hemiplegia of childhood, a rare disorder

For families of children born with alternating hemiplegia of childhood, the questions begin almost immediately: Will it get worse? How long do we have? What should we prepare for? The clearest answers yet are emerging and ...

May 17, 2026

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Tiny worms, with help from researchers, may hold key to treating rare childhood disease

A new worm model developed by Brown University researchers could play a key role in treating a rare genetic disease that causes paralysis in children and worsens with age. Developed in the lab of neuroscientist Anne Hart, ...

May 12, 2026

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Genome editing has corrected rare brain mutations in mice; could also help fight neurological diseases

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with neurological diseases.

Jul 21, 2025

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Mouse models for ultra-rare disorder could pave the way for nervous system gene editing therapies

Scientists at The Jackson Laboratory (JAX) have developed mouse models that survive premature death and enable pre-clinical testing of alternating hemiplegia of childhood (AHC), a devastating and sometimes fatal neurological ...

Jul 2, 2025

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Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate

Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden's rare, inherited immune deficiency. But it's not available to him.

Jun 21, 2024

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Oxygen therapy found to ameliorate abnormal movements in a rare childhood disease

Alternating hemiplegia of childhood is a severe neurodevelopmental disorder associated with a mutation in the ATP1A3 gene. It begins in infancy, causing symptoms that significantly impair quality of life. Young patients are ...

Feb 20, 2023

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Disease or Syndrome: Alternating hemiplegia of childhood

Medical news and research articles

Understanding alternating hemiplegia of childhood, a rare disorder

Tiny worms, with help from researchers, may hold key to treating rare childhood disease

Genome editing has corrected rare brain mutations in mice; could also help fight neurological diseases

Mouse models for ultra-rare disorder could pave the way for nervous system gene editing therapies

Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate

Oxygen therapy found to ameliorate abnormal movements in a rare childhood disease

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