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.