P35 Two-year clinical outcomes with fordadistrogene movaparvovec (FM) for Duchenne muscular dystrophy (DMD) and contextualization with external controls

We report the effect of FM on motor function at 2 y vs selected external control groups. Ambulatory subjects (n=16) with confirmed DMD received a single i.v. dose (2E14 vg/kg) of FM. An external control group was generated from a prediction model developed within the cTAP collaboration using large natural history databases. A second control group with similar baseline characteristics was constructed using 2-y data from subjects with at least 1 y of treatment in a recent null phase 2 trial.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: P. Shieh, R. Butterfield, F. Muntoni, E. Mercuri, J. Signorovitch, P. Schwartz, H. Li, M. Binks, T. McDonnell, K. Ryan, M. Delnomdedieu, Q. Shen, D. Levy, E. Smith Source Type: research