Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy.

ABSTRACT:

Objective

To understand the natural disease upper limb progression over 3 years of ambulatory and non-ambulatory patients with Duchenne muscular dystrophy (DMD) using functional assessments and quantitative magnetic resonance imaging (MRI) and to exploratively identify prognostic factors.

Methods

Forty boys with DMD (22 non-ambulatory and 18 ambulatory) with deletions in dystrophin that make them eligible for exon 53-skipping therapy were included. Clinical assessments, including Brooke score, motor function measure (MFM), hand grip and key pinch strength, and upper limb distal coordination and endurance (MoviPlate), were performed every 6 months and quantitative MRI of fat fraction (FF) and lean muscle cross sectional area (flexor and extensor muscles) were performed yearly

SUBMITTER: Lilien C 

PROVIDER: S-EPMC8528463 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature