Maryze's story - Part 8

On March the 29th Myozyme was officially approved in Europe. That special day for Pompe patients, I was at the funeral of a Pompe patient I knew very well. It was she who called me that day on November the 14th 1996, to ask if I had heard the news and what I thought of it. It was sad that she never could benefit from the treatment she too had been waiting for so long. Shortly after the FDA approved Myozyme as well. Slowly Pompe patients all over the world did start treatment. I know of one boy in Germany who was so affected that he didn’t meet the criteria for participation in the clinical trial in Rotterdam, but he was able to start commercial treatment with Myozyme just 2 weeks after the official approval.

A cake to celebrate the approval of Myozyme.

In spite of the approval the clinical trials on Myozyme in adults continued. The regulatory authorities had requested additional data that showed also efficacy of Myozyme in older Pompe patients. I personally admire these people who did continue going to Rotterdam and other trial sites in France and the USA to get their infusions not knowing if they received alpha-glucosidase or placebo, to do all the required tests and go through all the emotional feelings, even when they knew Myozyme was approved for the market already.

Over the years from 1996 till now, I collected a lot of documents, newspaper articles, publications etc on Pompe disease and related issues from all over the world. These 6 large files contain a lot of information on events and people not mentioned in the book ‘The Cure’. This book is a story of John Crowley and his family, but it's not the story of so many other Pompe patients in the world.