Gene therapy for krabbe disease: did it last?
NCT ID NCT06308718
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This study follows up on children with Krabbe disease who received a one-time gene therapy infusion (FBX-101) in earlier trials. Researchers will monitor safety and measure motor skills over time. Only 2 participants are enrolled, so results are very limited.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
FBX-101 (gene therapy)
What this could lead to
If successful, this follow-up could show whether a single gene therapy infusion provides lasting safety and motor benefits for children with Krabbe disease.
What could go wrong
This is a tiny observational follow-up (only 2 participants) of an already terminated trial. It cannot prove effectiveness and may not apply to other patients.
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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University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, 48109, United States