The treatment landscape for Pompe disease is undergoing a remarkable shift, especially within emerging markets, as increased awareness, regulatory advancements, and broader access to cutting-edge therapies begin to reshape the future for patients. Pompe disease is a rare and inherited genetic disorder caused by a deficiency in the enzyme acid alpha-glucosidase (GAA), leading to the accumulation of glycogen in muscle tissues, which results in severe muscle weakness and potentially life-threatening complications. Historically, this condition has been more commonly diagnosed and treated in developed regions such as North America and Europe. However, as Pompe disease emer...
