European regulators back new DMD gene therapy with key status
WHY IT MATTERS
SGT-003 now has regulatory backing in both the U.S. and Europe, which accelerates its development pathway and may bring a new treatment option closer to boys with DMD who currently have limited gene therapy choices.
European regulators have approved a special status for SGT-003, a new gene therapy treatment for Duchenne muscular dystrophy (DMD), a serious muscle disease that affects boys. This approval, called orphan drug designation, means the treatment is recognized as addressing a rare disease with few other options. The therapy is designed to help preserve muscle strength in boys with DMD.
Regulators in the European Union have granted orphan drug designation to SGT-003, a gene therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Solid Biosciences. The treatment candidate, already awarded orphan drug status in the U.S., aims to preserve muscle health in boys with DMD. This new designation, by the European Commission, is granted […] The post European regulators back new DMD gene therapy with key status appeared first on Muscular Dystrophy News .
ASK YOUR DOCTOR
If your son has DMD, ask your neurologist or muscular dystrophy specialist about SGT-003 and whether he might be eligible for upcoming clinical trials.