Gene therapy for devastating muscle disease tested in Real-World settings
NCT ID NCT07265232
Summary
This study is observing how well a gene therapy called Vesemnogene works for spinal muscular atrophy (SMA) in real-world settings in lower-income countries. Researchers will follow 15 patients with SMA who receive this one-time treatment to monitor their safety and see if they achieve important motor milestones like sitting or walking. The study aims to understand how this advanced treatment performs outside of controlled clinical trials, particularly for patients who cannot access other treatments.
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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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