Gene therapy for SMA tested in real-world settings of poorer nations
NCT ID NCT07265232
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing a gene therapy called vesemnogene lantuparvovec for spinal muscular atrophy (SMA) in low- and middle-income countries. Researchers will track safety and whether children reach motor milestones like sitting or walking. The trial involves 15 participants and aims to see if the treatment works outside of wealthy countries.
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 (a gene therapy)
What this could lead to
If successful, this could show that gene therapy for SMA is safe and effective in low-resource settings, potentially expanding access to treatment.
What could go wrong
This is a small, early real-world study with only 15 participants, so results may not apply broadly. Gene therapy carries risks like immune reactions and unknown long-term effects.
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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Tzu chi hospital
RECRUITINGJakarta, Indonesia, 14470, Indonesia
Contact Phone: •••-•••-•••• Email: •••••@•••••