New gene therapy tested for devastating infant muscle disease
Disease control
Ongoing
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 …
Phase: NA • Sponsor: Kun Sun • Aim: Disease control
Last updated Mar 23, 2026 15:16 UTC