The Pompe Disease Content Hub Channel
In this webinar, we delve into the advancements in Pompe disease treatment, particularly focusing on the progression from mouse models to clinical trials. Key developments include the testing and authorization of Lumizyme (formerly Myozyme) and other promising second-generation products. The discussion highlights Amicus’s work on AT-GAA, a two-part therapy combining miglustat and enzyme replacement therapy, aimed at improving treatment efficacy and accessibility. With the advent of newborn screening for Pompe disease, early intervention and better outcomes are expected. The Muscular Dystrophy Association continues to monitor long-term patient outcomes through comprehensive data collection.