P130 Two-year muscle MRI observations from a phase 1b trial of fordadistrogene movaparvovec (PF 06939926) for Duchenne muscular dystrophy (DMD)

Fordadistrogene movaparvovec (PF 06939926) is an adeno-associated virus serotype-9 (AAV9) gene-replacement construct containing a mini-dystrophin being developed for DMD, which aims to restore functional protein to muscle. Magnetic resonance imaging (MRI) is an important non-invasive tool that can be used for monitoring disease progression and can inform future functional changes. Here we present 2-yr quantitative MRI measurements from 16 ambulatory participants with a genetic diagnosis of DMD from a phase 1b, multicenter, single-arm, open-label trial (NCT03362502).
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research