Longitudinal Assessment of Muscle Involvement in Late-Onset Pompe Disease Using Quantitative MRI: A Prospective Cohort Study.
BACKGROUND
Late-onset Pompe disease (LOPD) is a progressive metabolic myopathy characterised by lysosomal glycogen accumulation and leading to secondary disruptions in autophagy and cellular energy metabolism. While enzyme replacement therapy (ERT) has improved outcomes, early detection remains critical, because irreversible muscle...
🗓️ 2026-06-01
📰 Publication: Journal Of Cachexia Sarcopenia And Muscle
Read MoreLongitudinal Assessment of Muscle Involvement in Late-Onset Pompe Disease Using Quantitative MRI: A Prospective Cohort Study.