Experimental drug aims to fix faulty kidney protein in rare genetic disease
NCT ID NCT05448755
Summary
This small, early-stage study tested whether daily injections of an experimental drug called ELX-02 are safe and can improve kidney health in adults with a specific genetic form of Alport Syndrome. The drug is designed to help the body produce a crucial protein that is missing due to a genetic error. Researchers measured changes in kidney structure and protein in the urine over 8 weeks of treatment.
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Contacts and locations
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Locations
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Great Ormond Street Hospital
London, WC1N 3JH, United Kingdom
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Monash Medical Center
Clayton, Victoria, 3168, Australia
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Royal Children's Hospital
Parkville, Victoria, 3051, Australia
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Royal Free Hospital
London, NW3 2QG, United Kingdom
Conditions
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