Massive study aims to unlock secrets of childhood nerve and muscle diseases

NCT ID NCT01568658

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

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This long-term study looks at children and adults with inherited nerve and muscle disorders that start early in life, like muscular dystrophy. Researchers will track symptoms over time and collect genetic samples from affected individuals, their family members, and healthy volunteers. The goal is to better understand these conditions and develop tools for future clinical trials.

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 better ways to measure disease progression and find genetic causes, helping design future treatments.
What could go wrong: This is an observational study, not testing a treatment. It may not directly lead to new therapies, and results depend on long-term participation.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

hereditary neurological disease hereditary neuromuscular disease hereditary peripheral neuropathy hereditary spastic paraplegia muscular dystrophy myopathy neuromuscular disease

As listed by the trial registrant

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

HEREDITARY SPASTIC PARAPLEGIAS INHERITED NEUROMUSCULAR CONDITIONS INHERITED NEUROPATHIES MUSCLE MYOPATHIES MUSCULAR DYSTROPHIES

Contacts and locations

Locations

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

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