The 1998 scene

I've been looking back through my archive of 1998. It's difficult to convey in words the sense of momentum, of excitement, of a community waking up. I have hundreds and hundreds of pages of Pompe emails from this time (they were all bundled into a Word document at some point) - and that is only the archive from my work email address. Along with the hard copy archive, they build up a picture of almost feverish activity. Patients, scientists and industry all communicating, sharing information, building relationships - motivated by a common goal.  Something truly wonderful happened in those few years, starting in 1998.  Although I wish I had never heard of Pompe disease, I also that feel, strongly, that it was a privilege to be part of that time. 

Once again, I must make special mention of Randall and Marylyn House. Their prime motivation, understandably, was to find a treatment for Tiffany. But they did not stop there. They could have developed their connection with Pharming, went home and not bothered to return anyone else's calls. But they didn't.  Randall spent a great deal of time and resources travelling the world attending conferences and encouraging people to start their own Pompe patient groups.  Marylyn continued to run the AMDA, grouping together the US patient community, putting out newsletters and organising conferences. They didn't have to do any of those things. But they did. And they made a difference - not just for Tiffany but for everyone.

I was soon to get the opportunity to see the work of the AMDA at first hand, when I was invited to speak at their third conference, organised in Bethesda, Maryland USA in December 1998.