International Pompe Association

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Background

Throughout the second half of 2008 board members of the International Pompe Association worked closely with the biotech company, Genzyme Corporation, and expert treating physicians to monitor the global Myozyme supply situation. Our main objective was to prepare a plan of action to modify patient demand should inventories become so tight as to jeopardise the treatment of the most vulnerable infants, children and adults within the Pompe community.

As it turned out, the disruption to supply was restricted to a single skipped infusion in early 2009 for most adults. However the IPA was aware that there were noticeable effects from this delay in treatment and so we decided to survey a sample of the patient community to make an assessment of the true impact. 

Given the amount of time and effort required to prepare the Myozyme Stakeholders Working Group (MSWG) guidelines, and also the preparation of a global communication plan, we hoped to assess their quality through the same survey.

The questions presented grew from the original concept to 38 in number, very few questions were compulsory and respondents were at liberty to skip any questions that they were not comfortable answering, could not remember, or did not apply.

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