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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Kunming Hope of Health Hospital
Kunming, Yunnan, 650200, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can gene therapy help babies with SMA reach milestones? a Real-World review
- School transitions put to the test for children with a rare muscle disease
- Horseback therapy may boost movement and breathing in kids with Muscle-Weakening disease
- Newborn screening study aims to catch rare diseases at birth
- Higher dose of spinraza tested in SMA patients who already tried risdiplam
- Video games as therapy: VR shows promise for children with muscle disorders