DMD – THERAPY: P.284 ATL1102 treatment improves PUL2.0 in non-ambulant boys with Duchenne muscular dystrophy compared to a natural history control

Abstract

ATL1102, an antisense drug which targets CD49d, an immune cell adhesion, survival and activation molecule, is being evaluated in an open label study in nine adolescent non-ambulant patients with Duchenne muscular dystrophy (DMD) ACTRN12618000970246. The nine patients, 12 to 18 years old (mean 14.9 (SD 2.1) years), were dosed with ATL1102 once weekly at 25mg s.c. for 24 weeks and were assessed using the Performance of the Upper Limb Module (PUL2.0). The data of the ATL1102-treated patients (8 of 9 on corticosteroid) was compared with the 24-week PUL2.0 data of 20 patients (mean age 15.61 (SD 2.02) years; 19 on corticosteroid) from a natural history database of patients in Rome, Italy.