Gene therapy hope for SMA patients in low-income countries
NCT ID NCT07265232
First seen Jan 09, 2026 · Last updated May 16, 2026 · Updated 24 times
Summary
This study is testing a gene therapy called vesemnogene lantuparvovec for people with spinal muscular atrophy (SMA) in low- and middle-income countries. The goal is to see how well it works and how safe it is in everyday medical practice. About 15 participants will be followed for up to 5 years to check for side effects and improvements in motor skills like sitting or walking.
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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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Contacts and locations
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Locations
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Tzu chi hospital
RECRUITINGJakarta, Indonesia, 14470, Indonesia
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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