New gene therapy aims to help babies with deadly muscle disease
Disease control
Not yet recruiting
This early-stage study tests a new medicine called SKG0201 in 11 babies with a severe muscle disease (spinal muscular atrophy type I). The goal is to see if it is safe and if it can improve muscle function. Babies must be under 6 months old to join.
Phase: PHASE1, PHASE2 • Sponsor: Lanyue Biotech (Hangzhou) Co., Ltd. • Aim: Disease control
Last updated May 05, 2026 11:53 UTC