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Scientists launch study to unravel RNA's role in rare brain diseases

NCT ID NCT04394871

Recruiting now Knowledge-focused Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study aims to learn how the binding of RNA with DNA (called R-loops) is linked to amyotrophic lateral sclerosis type 4 (ALS4) and other inherited neurological disorders. Researchers will observe up to 330 people aged 5 and older, including those with ALS4, related conditions, and healthy volunteers. Participants undergo tests like MRI, blood draws, and muscle strength assessments over up to 3 years, but no experimental treatment is given.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

Aicardi-Goutieres syndrome amyotrophic lateral sclerosis type 4 progressive external ophthalmoplegia with mitochondrial DNA deletions, autosomal dominant 2 spinocerebellar ataxia, autosomal recessive, with axonal neuropathy 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AMYOTROPHIC LATERAL SCLEROSIS TYPE 4 INHERITED NEUROLOGICAL DISORDERS OF RNA PROCESSING

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••