One-of-a-Kind drug created for single Patient's rare disease
NCT ID NCT07226297
Summary
This study is testing a custom-made drug designed specifically for one person with a rare, inherited nerve disorder called Charcot-Marie-Tooth disease type 2D. The drug aims to slow or stop the progression of the disease by targeting the patient's unique genetic mutation. Researchers will closely monitor the patient's motor skills, safety, and quality of life over two years to see if the treatment works.
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Contacts and locations
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Locations
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UTHealth Houston
Houston, Texas, 77030, United States
Conditions
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