The 2024 Pompe Survey Report from Erasmus MC is the first to publicly bring together what patients themselves report about their symptoms, quality of life and treatment. For example, 81% of participants reported muscle cramps and 76% reported mobility problems, while treatment is gradually shifting from first-generation enzyme replacement therapy to newer therapies.
The IPA/Erasmus MC Pompe Survey has been running since 2002 and is a unique, independent source of long-term data on living with Pompe disease. With new treatments becoming available, continuing to monitor their impact on patients' lives remains important.
