Muscle MRI Findings in Childhood/Adult Onset Pompe Disease Correlate with Muscle Function.

ABSTRACT:

Objectives

Enzyme replacement therapy has shown to be effective for childhood/adult onset Pompe disease (AOPD). The discovery of biomarkers useful for monitoring disease progression is one of the priority research topics in Pompe disease. Muscle MRI could be one possible test but the correlation between muscle MRI and muscle strength and function has been only partially addressed so far.

Methods

We studied 34 AOPD patients using functional scales (Manual Research Council scale, hand held myometry, 6 minutes walking test, timed to up and go test, time to climb up and down 4 steps, time to walk 10 meters and Motor Function Measure 20 Scale), respiratory tests (Forced Vital Capacity seated and lying, Maximun Inspiratory Pressure and Maximum Expiratory Pressure), daily live acti

SUBMITTER: Figueroa-Bonaparte S 

PROVIDER: S-EPMC5053479 | biostudies-literature | 2016

REPOSITORIES: biostudies-literature