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

Glycogen Storage Disease Type II Clinical Trial

Official title:
Effect of Motor Development, Motor Function and Electrodiagnostic Characteristic of IOPD Under ERT

Clinical Trial Summary

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

Clinical Trial 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

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02761421
Study type Observational
Source Taipei Veterans General Hospital, Taiwan
Contact chihjou lai
Phone 886-28712121
Email irbopinion@vghtpe.gov.tw
Status Recruiting
Phase N/A
Start date January 2016
Completion date December 2016
View Details
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