Hope for rare nerve disease: new drug targets toxic sugar buildup

NCT ID NCT05397665

Completed Disease control Sponsor: Applied Therapeutics, Inc. Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

neuronopathy, distal hereditary motor, autosomal recessive 8

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEREDITARY NEUROPATHY CAUSED BY SORD DEFICIENCY

Contacts and locations

Locations

Axon Clinical, s.r.o.

Prague, Prague, 150 00, Czechia
Fondazione IRCCS Istituto Neurologico "Carlo Besta"

Milan, Milano, 20133, Italy
Hassman Research Institute

Berlin, New Jersey, 08009, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
University College of London

London, London, WC1E 6BT, United Kingdom
University of Colorado

Aurora, Colorado, 80045, United States
University of Iowa

Iowa City, Iowa, 52242, United States
University of Miami

Coral Gables, Florida, 33146, United States
University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States
University of Rochester

Rochester, New York, 14642, United States