New hope for rare bone marrow failure: experimental drug EXG34217 enters human testing
NCT ID NCT04211714
First seen Nov 01, 2025 · Last updated May 25, 2026 · Updated 33 times
Summary
This early-stage study tests a new drug called EXG34217 in 12 people aged 12 and older who have bone marrow failure due to telomere biology disorders. The main goal is to check if the drug is safe and tolerable. Researchers will also look for improvements in blood cell counts and telomere length.
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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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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
Conditions
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