Scientists launch deep dive into genetic brain diseases to unlock RNA mysteries

NCT ID NCT04394871

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

First seen Dec 12, 2025 · Last updated May 15, 2026 · Updated 24 times

Summary

This study aims to understand 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 blood draws, muscle strength assessments, and imaging over up to 3 years. No treatment is given; the goal is to gather knowledge about disease progression and biomarkers.

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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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Contacts and locations

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Study contacts

Contact

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

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

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States

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

Conditions

Explore the condition pages connected to this study.

AMYOTROPHIC LATERAL SCLEROSIS TYPE 4 INHERITED NEUROLOGICAL DISORDERS OF RNA PROCESSING