Condition: Spinal Muscular Atrophy · Sponsor: GeneCradle Inc
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
This description comes directly from the study's public registry record.
GeneCradle, Inc. China · 86-13501380583 · ind@bj-genecradle.com
Always discuss trial participation with your own doctor first.
| Peking University， First Hospital, Department of Pediatrics | Beijing, China | Recruiting |
| Bayi Children's Hospital, Seventh Medical Center, PLA general hospital | Beijing, China | Recruiting |
| Shengjing Hospital of China Medical University | Shenyang, China | Recruiting |
| Children's Hospital of Soochow University | Suzhou, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT05824169