New gene therapy tested for devastating infant muscle disease
NCT ID NCT06191354
Summary
This early-stage study is testing a new gene therapy called SKG0201 in infants with spinal muscular atrophy type 1, a severe genetic muscle-wasting disease. Researchers are checking if the treatment is safe and looking for early signs that it might help babies survive longer and improve their movement. The study involves 12 very young infants (6 months or younger) who haven't received other SMA treatments.
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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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