Experimental drug targets toxic sugar to halt rare nerve disease
NCT ID NCT05397665
Summary
This study is testing whether a drug called AT-007 can help control a rare, inherited nerve disease called SORD deficiency. The drug aims to lower high levels of a harmful sugar alcohol (sorbitol) in the body, which is thought to damage nerves and cause walking difficulties. About 56 adults with the condition will take either AT-007 or a placebo daily for up to two years to see if the drug improves their walking speed and other measures of function.
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Contacts and locations
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Locations
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Axon Clinical, s.r.o.
Prague, Prague, 150 00, Czechia
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Fondazione IRCCS Istituto Neurologico "Carlo Besta"
Milan, Milano, 20133, Italy
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Hassman Research Institute
Berlin, New Jersey, 08009, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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University College of London
London, London, WC1E 6BT, United Kingdom
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University of Colorado
Aurora, Colorado, 80045, United States
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University of Iowa
Iowa City, Iowa, 52242, United States
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University of Miami
Coral Gables, Florida, 33146, United States
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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University of Rochester
Rochester, New York, 14642, United States
Conditions
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