New gene therapy hopes to save babies with fatal muscle disease
Disease control
Ongoing
This study tests a new gene therapy called SKG0201 in 12 infants with spinal muscular atrophy type 1, a severe muscle-weakening disease. The goal is to see if the treatment is safe and can help babies survive and move better. All participants are under 6 months old and receive a …
Phase: NA • Sponsor: Kun Sun • Aim: Disease control
Last updated May 11, 2026 20:38 UTC