Gene therapy trial for rare nerve disease halted early
NCT ID NCT05394064
First seen Oct 31, 2025 · Last updated May 10, 2026 · Updated 27 times
Summary
This study tested a one-time gene therapy called SBT101 for adults with adrenomyeloneuropathy (AMN), a rare nerve disease that affects walking. The treatment was given as a single injection into the spine. The study was terminated early after enrolling only 8 people, so results are limited. The goal was to see if the therapy was safe and could slow disease progression.
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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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Locations
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Amsterdam UMC
Amsterdam, Netherlands
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University of Massachusetts Chan Medical School
Worcester, Massachusetts, 01655, United States
Conditions
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