Mayo clinic launches quest for custom drugs for rare genetic diseases
NCT ID NCT05236595
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study at Mayo Clinic aims to find people with rare genetic diseases who might benefit from a custom-made drug designed to target their specific genetic change. Researchers will collect samples like blood and skin biopsies to develop and test potential treatments. The goal is to eventually create new therapies for conditions that currently have no good options.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
individualized drug matching per genetic disease
What this could lead to
If successful, this could lead to new personalized treatments for people with rare genetic diseases that currently have no options.
What could go wrong
This is an early-stage research study focused on identifying candidates and developing drugs, not testing them yet. Many potential drugs may not work or be safe.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Mayo Clinic Florida
Jacksonville, Florida, 32224, United States
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Mayo Clinic Rochester
Minneota, Minnesota, 55905, United States
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Mayo Clinic in Arizona
Scottsdale, Arizona, 85259, United States