Mayo clinic launches quest for custom drugs for rare genetic diseases
NCT ID NCT05236595
First seen Jan 22, 2026 · Last updated May 15, 2026 · Updated 12 times
Summary
This study aims to find 50 people with rare genetic diseases that currently have no good treatment. Researchers will collect blood and tissue samples to understand each person's unique genetic change. They hope to develop personalized drugs, called antisense oligonucleotides, that target the specific genetic cause. The study focuses on learning about the disease and testing new treatments, not on providing a cure.
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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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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
Conditions
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