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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Saturday 16 January 2010

2001 continued

Things seemed to be on a pretty even keel, even if progress was not quite as fast as we might have liked.

Then came the news of the Genzyme/Novazyme merger in August 2001. I have already dealt with the Novazyme story in earlier posts and don't propose to re-hash the whole sorry story again here.

This was swiftly followed by the news that Pharming had gone into receivership.  Thankfully, as noted before, they survived to fight another day. Genzyme acted to ensure that there was a continued supply of the rabbit enzyme for those patients receiving it, though it was clear that they would now be even more keen in transitioning those patients to the CHO enzyme as soon as possible.

Nevertheless, it raised a lot of concerns - we appeared to have been on one course and now everything was, once again, up in the air. Thankfully, an AMDA patient meeting was scheduled for September 2001, in San Antonio, Texas. As well as being the first opportunity for may US patients to get together, Genzyme had undertaken to participate and answer questions.

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