Personalized study for rare genetic diseases using genetic “target” drugs
Part of Genetic & congenital clinical trials.
This study looks for rare genetic conditions where a personalized genetic medicine (an antisense drug) could potentially work. If the team finds a “targetable” DNA or RNA change in your family, you may be followed to see how the best therapy could be planned.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have care at Mayo Clinic or another health system with a unique medical ID
- You (or your legal representative) can understand the study and sign consent
- A genetics professional has found a likely disease-causing genetic change from a DNA test
- Your genetic change must be the kind that antisense medicines may be able to target
- You have a biological family member already enrolled in the study
- You can travel to a Mayo Clinic site for treatment if a therapy is developed
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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