Hope for rare nerve disease: new drug targets toxic sugar buildup
NCT ID NCT05397665
First seen Nov 01, 2025 · Last updated May 17, 2026 · Updated 21 times
Summary
This study tests an experimental drug called AT-007 in people with a rare inherited nerve disorder (SORD deficiency) that causes weakness and walking problems. The drug aims to lower harmful sorbitol levels in cells, which build up due to the genetic defect. About 56 adults aged 18-55 will receive either AT-007 or a placebo for up to 24 months to see if it improves walking speed and reduces sorbitol.
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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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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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