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Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT

2016-05-04 21:53:21 | BioPortfolio

Summary

To investigate the motor development, motor function and electrodiagnostics presentation in IOPD under ERT.

Description

Background

Pompe disease is an autosomal recessive disease, enzyme replacement therapy (ERT) and new born screen (NBS) had been initiated in Taiwan since 2008. However, residual muscle weakness were noted. Decrease in amplitude of nerve compound muscle action potential (CMAP) and increase spontaneous activity were reported before the imitation of ERT. However, the motor development, motor function and electrodiagnostic presentation which were important in further direction of treatment and rehabilitation program arrangement in infantile Pompe disease (IOPD) under ERT is lacking.

Aim

To investigate the motor development, motor function and electrodiagnostic presentation in IOPD under ERT.

Method

This is an observational, prospective, longitudinal, follow-up study. Motor development was assessed by Alberta Infant Motor Scale (AIMS) and Peabody Developmental Motor Scales, Second Edition (PDMS II); motor function was assessed by Pediatric Evaluation of Disability Inventory (PEDI) and Gross Motor Function Measure (GMFM). Electrodiagnosis studies include nerve conduction study (NCS) and electromyography (EMG).

Expect Effects

Obtain the characteristics of motor development, motor function and electrodiagnosis presentation of IOPD under ERT, and the relation between motor development, motor function and electrodiagnosis presentation.

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Conditions

Glycogen Storage Disease Type II

Intervention

observation study

Location

Taipei Veteran General Hospital : Taipei City, Taiwan 11217, R.O.C.
Taipei
Taiwan
11217

Status

Recruiting

Source

Taipei Veterans General Hospital, Taiwan

Results (where available)

View Results

Links

Published on BioPortfolio: 2016-05-04T21:53:21-0400

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