Scientists track how a single gene causes two brain diseases

NCT ID NCT01925196

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study followed 50 adults with a C9ORF72 gene mutation that can cause ALS or frontotemporal dementia. Over three years, researchers measured changes in strength, thinking, memory, and behavior using brain scans, spinal fluid tests, and other assessments. The goal was to understand how symptoms evolve and find biomarkers that could help detect or track these diseases in the future.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

What this could lead to

If successful, this study could identify biomarkers to help detect or measure C9ORF72-related ALS and FTD, aiding future research.

What could go wrong

This is an observational study with only 50 participants, so findings may not apply to everyone. It does not test any treatment.

Disclaimer Read more

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.

amyotrophic lateral sclerosis frontotemporal dementia Frontotemporal Lobar Degeneration motor neuron disorder

As listed by the trial registrant

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

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States