New gene therapy tested on infants with devastating muscle disease
NCT ID NCT06191354
Summary
This early-stage study is testing a new gene therapy injection called SKG0201 in babies with spinal muscular atrophy type 1, a severe genetic muscle-weakening condition. Researchers are giving increasing doses to 12 infants to check if the treatment is safe and to see early signs of whether it might help. They'll monitor survival, breathing support needs, and whether babies reach developmental milestones like head control and movement.
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Contacts and locations
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Locations
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National Children's Medical Center, Children's Hospital of Fudan University
Shanghai, China
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West China Sencond Hospital, Sichuan University / West China women's and children's Hospital
Chengdu, Sichuan, China
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Xinhua Hospital Affiliated To Shanghai Jiao Tong University School Of Medicine
Shanghai, 200092, China
Conditions
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