Hope for rare nerve disease: new drug targets toxic sugar buildup
NCT ID NCT05397665
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests an experimental drug called AT-007 (Govorestat) in 56 adults with SORD deficiency, a rare genetic condition that causes nerve damage and muscle weakness. The drug aims to lower high levels of sorbitol, a sugar that builds up and harms nerves. Researchers will measure if it improves walking speed and other daily functions over up to 24 months.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Govorestat (AT-007), an aldose reductase inhibitor
What this could lead to
If successful, this could lead to the first approved treatment for SORD deficiency, potentially slowing or improving nerve damage and mobility.
What could go wrong
This is a mid-stage trial with only 56 participants, so results may not apply to everyone. The drug may not improve symptoms enough to be meaningful, and side effects are still being studied.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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