Decoding the muscle transcriptome of patients with late-onset Pompe disease reveals markers of disease progression
A Monceau, RG Nath, X Suárez-Calvet, O Musumeci… - Brain, 2024 - academic.oup.com
Late-onset Pompe disease (LOPD) is a rare genetic disorder caused by the deficiency of
acid alpha-glucosidase leading to progressive cellular dysfunction owing to the …
acid alpha-glucosidase leading to progressive cellular dysfunction owing to the …