Ataluren use in nonsense mutation Duchenne muscular dystrophy: patient information from the STRIDE Registry

Lay abstract Duchenne muscular dystrophy (DMD) is a rare disorder that causes progressive muscle degeneration and weakness. In around 10–15% of boys with DMD, the disorder is caused by a nonsense mutation in the DMD gene, so-called nonsense mutation (nm)DMD. Ataluren is a first-in-class, orally bioavailable treatment for patients with nmDMD. The STRIDE Registry is an ongoing, multicenter, observational, post-approval safety study of ataluren in patients with nmDMD, which was set up to gather data on patterns of use and long-term outcomes in real-world clinical practice. The STRIDE Registry is the first drug registry for DMD patients and represents the...