Pompe disease is a rare genetic disorder that presents a spectrum of severity, with varying rates of progression and ages of onset. Symptoms can appear anywhere from infancy to late adulthood, with earlier onset generally associated with more rapid progression and increased severity. At all ages, the disease is marked by skeletal muscle weakness, leading to mobility issues and respiratory complications. Pompe disease prevalence spans different ethnic groups worldwide, with an incidence rate of approximately 1 in 40,000 births in the United States and the Netherlands. According to DelveInsight, there were about 13,000 total prevalent cases of Pompe disease in 2023, wit...
