Gene therapy trial aims to halt Muscle-Wasting disease
NCT ID NCT06288230
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a gene therapy called vesemnogene lantuparvovec in about 20 people with spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The therapy delivers a working copy of the SMN1 gene to help muscles work better. The main goal is to check safety, but researchers will also see if it improves motor skills like sitting or walking.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
vesemnogene lantuparvovec (gene therapy)
What this could lead to
If successful, this could provide a new treatment option that improves motor function and slows disease progression in people with spinal muscular atrophy.
What could go wrong
This is a small, early-phase trial (Phase 1/2) focused on safety, so it is too soon to know if the therapy works. There may be risks from the gene therapy or the viral delivery system.
Disclaimer
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This is a summary of
the original study
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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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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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Kunming Hope of Health Hospital
Kunming, Yunnan, 650200, China