Drs. Reuser and van der Ploeg started the first clinical trial with enzyme replacement therapy in January 1999 in Rotterdam, the NL.  Not only was ERT (enzyme replacement therapy) a novelty therapy for this rare disease, but so was the method of producing the enzyme.  It was derived from the milk of transgenic rabbits. This method and another enzyme therapy (CHO derived enzyme) that was developed at Duke University in the US, are the forerunners of Myozyme an enzyme replacement for Pompe disease that is produced by Genzyme Corporation.  Myozyme was approved in the US by the FDA and in Europe by the EMEA in April 2006. Today Pompe patients the world over are receiving therapy that grew from the research efforts of a small community of dedicated scientists.  Drs. Reuser and van der Ploeg were and are leaders in the Pompe field and should be honored as true advocates of patients with a rare disease.

Thank you Dr. Reuser and Dr. van der Ploeg.

Tiffany House