What is it about?

We propose the "Gait/Stair/Gowers/Chair" orGSGC scale as a new simple outcome test in Pompe adult patients , the test is easy, fast and reliable

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Why is it important?

GSGC has been validated by a multicentric stud in a series of late-onset Pompe disease patients in several Italian centers, and is now in use in US trials for Enzyme Replacement Therapy.


This scale will be increasingly used with our license in the US and Europe for further trials in late onset Pompe patients.Diet might be a useful add on to ERT was found in a recent article published in EJTM, and exercise added according to a new PRO.

Prof Corrado Angelini
Universita degli Studi di Padova Polo Bibliotecario di Scienze Farmacologia e Scienze Farmaceutiche

Read the Original

This page is a summary of: New motor outcome function measures in evaluation of Late‐Onset Pompe disease before and after enzyme replacement therapy, Muscle & Nerve, May 2012, Wiley,
DOI: 10.1002/mus.23340.
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