What the development of Myozyme meant for patients, their families, and their community

What the development of Myozyme meant for patients, their families, and their community

Posted On: 07/04/2022

The story of the UK Pompe community’s involvement in the development of enzyme replacement therapy for Pompe disease is the subject of an academic publication.

Japanese researcher Koichi Mikami interviewed many of the people involved, including the Dutch scientists who pioneered the treatment and PSN trustees Allan Muir and Kevin O’Donnell. Despite being an academic, peer-reviewed publication, it is very readable, as well as thorough and balanced.

You can read it yourself from the following link: Download