Massive gene hunt aims to solve medical mysteries for thousands with rare diseases

NCT ID NCT02743845

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

Summary

This study at Boston Children's Hospital is enrolling up to 10,000 people with rare or undiagnosed conditions, along with their family members. Researchers will analyze DNA samples to find the genetic causes of these illnesses. The goal is to improve diagnosis and understanding of rare diseases, which could eventually lead to better treatments.

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 research could lead to better diagnosis and, eventually, new treatments for rare genetic diseases.

What could go wrong

This is an observational study, not a treatment trial. Finding a genetic cause does not guarantee a cure or therapy, and results may take years.

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.

Rare Diseases

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

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