Condition: Spinal Muscular Atrophy (SMA) · Sponsor: Lantu Biopharma
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA. The specific objectives are: * To determine clinical effectiveness of Vesemnogene lantuparvovec therapy for SMA as evaluated by developmental gross motor milestone and survival. * To describe the safety profile of Vesemnogene therapy for SMA as evaluated by adverse events reporting and laboratory tests, and monitoring of Adverse events of special interest.
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| Tzu chi hospital | Jakarta, Indonesia, Indonesia | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07265232