New Treatment Developments for Pompe Disease
A Letter from Allan Muir, Chair, Pompe Support Network
Posted On: 30/09/2020
I’ve been wanting to write this article for some time but was slightly daunted by the number of potential treatments currently under study; some paused due to Covid-19, and a few reopening their clinical trial sites. So, it was a wonderful surprise to receive notification of a paper published recently on this very subject. The paper’s lead author is the highly respected Nina Raben who has worked for many years to understand the underlying biochemistry of Pompe disease and it was her work that led to the Pompe mice models that were used to prove the principal of Enzyme Replacement Therapy for Pompe back in the mid to late 1990s.