Publications

for Patients

Flying with a Ventilator in the United States (737.75 kB)

A resource for patients in the US, or for those traveling to the US who are dependent on artificial ventilation.

This is a brochure compiled by Genzyme in an effort to aid patients in overcoming their fear of needles, an integral part of the Myozyme infusion process.

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Pompe’s Disease - World success with a setback: laborious production of the medicine

Kees Vermeer; translation by Gezinus Wolters
Erasmus Monitor, Erasmus Medical Centre, Rotterdam, April/May 2004

"Research at the Erasmus Medical Centre in Rotterdam on Pompe’s Disease has resulted in a world success: it has been proven now that the so-called enzyme replacement therapy (ERT) works. But the road to that success has known many setbacks."

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Summary of the LOTS Clinical Trial (148.13 kB)

This is a summary of the Late-Onset Treatment Study (LOTS). The study was the irst and only placebo-controlled clinical study in Pompe patients. 90 patients were enrolled between September 2005 and September 2007.

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