Neuropediatrics
Evaluation of Experienced Clinical Events in Pompe Disease Based on Real-life Data.
Fehime Erdem Karapınar, Havva Yazıcı, Merve Yoldaş Çelik, Ayşe Yüksel Yanbolu, Cenk Eraslan, Asude Durmaz, Ayça Aykut, Ebru Canda, Zülal Ülger Tutar, Sema Kalkan Uçar, Ertürk Levent, Eser Yıldırım Sözmen, Mahmut Çoker
Published: 202610.1055/a-2777-2932
Abstract
Pompe disease is a rare lysosomal storage disorder with a wide clinical spectrum ranging from infantile-onset Pompe disease (IOPD) with early severe cardiomyopathy to late-onset Pompe disease (LOPD) with progressive muscle weakness. This study aimed…
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