The Duke results

Although the results were now widely known, it is worth noting that March 2001 saw the publication of the results of the Duke University trial led by YT Chen, using the Synpac enzyme. There was the inevitable press release from Genzyme/Pharming and a paper published in Genetics in Medicine.  Only the abstract is available free unfortunately.

This largely (with the caveat previously mentioned) confirmed the results from the Rotterdam trail.

It was also a real achievement for Yuan-Tsong Chen and his co-workers. Known to all as 'YT', Chen was (still is) a well known and well-liked figure amongst US patients. This was partly because his interest in the glycogen storage diseases extended to types I and III, the liver-based GSDs. These are very different from Pompe and, thankfully, more treatable. YT had the reputation of being a diligent and committed doctor, as well as a good scientist.

One name missing from the list of authors for the Duke paper was Johan van der Houe. He had been a collaborator in the earlier Chen papers and retained a close interest in the subject. He had returned to his native Belgium, where he was involved in the clinical trials. I had met Johan at a number of meetings of the years - he was, in fact, one of my earliest and most helpful scientific contacts. His commitment and contribution deserves to be noted and this seems like a good place to do so.