Before we get into the 2003 big picture, can I just step aside for a moment and mention something of huge significance for me. This was the year that, for the very first time, there was a UK clinical trial of ERT. This was at The Willink Centre in Manchester, under Dr Ed Wraith and co-workers. This was a dream come true and added to a growing feeling that my own part in the story, such as it was, was coming to a logical end.
2003 was again a year of solid progress. In retrospect, we had moved pretty clearly to a situation where we knew that ERT would be available. For many people, this was even more difficult than wondering if it would be available. It was now a waiting game, not a hoping game. In particular, many adult patients were wondering when it would be their turn to feature in a trial.
This led to occasional tensions. The IPA were in regular contact with Genzyme, yet much of this was under strict terms of confidentiality. This meant that information could not be passed on to the patients, who were - understandably - restive.
Nevertheless, a series of regular updates held things together.
Issue 10 of the Pompe's Bulletin came out in February, bringing people up to date on a whole range of things, including the Manchester trial.
There were regular Pompe Program updates (contents agreed between the IPA and Genzyme) of progress.
These were released on February 18, March 19 (Genzyme press release), September 10 (press release)
The latter brought the first news of adult onset trials and of 'Special Access' for patients who did not fit into clinical trials but who were in demonstrable need. This was a mark of Genzyme's success in ramping up production - they could not have made such promises without absolute confidence in supplies of enzyme. From now on, alpha-glucosidase would be much more widely available, in advance of formal commercialisation.
As you can imagine, having been aware of developments through regular contact with Genzyme, it was a great feeling of relief to have them out in the public domain. It was recognised though that something bigger and public was needed to signal progress to patients. That something was the biggest and best Pompe event so far - the 2003 IPA Conference at Heidelberg in Germany.