Gene therapy hope for 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 treatment aims to improve survival and motor skills by delivering a working gene. Researchers are checking safety and how well the therapy …
Phase: NA • Sponsor: Kun Sun • Aim: Disease control
Last updated Jun 27, 2026 07:58 UTC