New hope for children with Rapid-Aging disease as experimental drug enters next testing phase
NCT ID NCT06775041
Summary
This study aims to find the best dose and check the safety of a new drug called progerinin for children with Hutchinson-Gilford progeria syndrome, a rare and severe rapid-aging disease. Ten participants will either take the standard treatment alone or combined with progerinin. The main goals are to see how the body processes the drug and to monitor for any side effects.
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Contacts and locations
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Conditions
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