Dr J C Pompe

Dr J C Pompe
Discoverer of Pompe disease

About this blog

What you can read here is the story of the development of enzyme replacement therapy (ERT), the first effective treatment for Pompe disease. It is an incredible story, rich with events, characters and science. Above all, it is the story of an international community of scientists, doctors, patients and companies, working together towards a common goal.

It is not a story that features in Geeta Anand's book, The Cure , or the film based on it, Extraordinary Measures despite the fact that they are ostensibly about the development of ERT for Pompe ( you can link straight to the relevant articles covering the events described in the book and film here, here and here).

This blog represents my small attempt to set the record straight and to give the story back to its rightful owners - the international Pompe community. It is written here in roughly chronological order i.e. you'll need to start at the bottom of the April 2009 archive page and work your way up.

It is also a personal account and, although I've tried to make it as objective as possible, there is an inevitable degree of subjectivity. For that reason I have included contributions from other members of the worldwide Pompe community and would be delighted to receive more. Feedback is also welcome.

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Sunday, 17 January 2010

A small personal note on 2001

I can't end the account of the events of 2001 without noting a small but significant personal change for me. I stood down as the Pompe representative on the AGSD-UK Executive. Partly because I was just worn out with it really. Partly because I had always seen my involvement as finite - I would do it until a treatment was available. That day was was now not so far off.  And partly again because I was in the happy position of having a good successor in Allan Muir.

I've already sung Allan's praises once, so I won't embarrass him by doing it again. Except to note that he is one of those people who seems to radiate calm, when everyone else is in a flap. A quality I can only admire.

From now on, the rest of this blog (at least the bits written by me) will be somewhat detached from the UK scene. I retained my involvement with the IPA for another couple of years though (before cunningly diverting that to Allan too).

I therefore only have first-hand experience of two more years - 2002 and 2003.

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