Enzyme replacement therapies in adults with Pompe disease: from trials to real-world data.
van der Beek NAME, Potters LH, Schoser B
Current opinion in neurology, 2025 Jun 06
Abstract
To review the clinical trial results and emerging real-world data of two new enzyme replacement therapies (ERTs) for late-onset Pompe disease and to compare these effects in the context of what has been achieved over the last two decades in advancing care for Pompe disease.