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 spinal muscular atrophy type I, a severe genetic condition that causes muscle weakness. The main goals are to check if the treatment is safe and to see if it can help babies survive and develop motor ski…
Phase: PHASE1, PHASE2 • Sponsor: Lanyue Biotech (Hangzhou) Co., Ltd. • Aim: Disease control
Last updated Jun 16, 2026 12:52 UTC