A milestone

Following the release of the Pharming/Genzyme results, we held an IPA telephone conference on 20 March 2000. This marked an important milestone.

One of the items concerned the extension of the trial mentioned in the press release - round 8 babies would be treated at Essen, in Germany. At the moment they had no-one and were actively recruiting. If I can add a personal note, this meant that, after 7 years of speaking with distressed parents from all over the globe, such parents could now be given something a bit better than sympathy. There was now a treatment, it had been shown to be of some benefit - and there were places available on a clinical trial right now. I felt a weight lifting. How much more must that have meant to doctors giving the diagnosis and the parents who received it.

We also agreed to co-operate with the Rotterdam team in drawing up a database of Pompe patients and their clinical progress and symptoms.

We were now waiting for two things - the results from the Duke/Synpac trial and the plan to commercialise the alpha-glucosidase product.  The latter turned out to be more complex than we could have imagined at the time.