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- Adjunctive β2‐agonists reverse neuromuscular involvement in murine Pompe disease Journal Articles
- Aerobic training as an adjunctive therapy to enzyme replacement in Pompe disease Journal Articles
- Combined aerobic exercise and enzyme replacement therapy rejuvenates the mitochondrial–lysosomal axis and alleviates autophagic blockage in Pompe disease Journal Articles
- Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α Journal Articles
- Novel GAA sequence variant c.1211 A>G reduces enzyme activity but not protein expression in infantile and adult onset Pompe disease Journal Articles
- Nutritional co-therapy with 1,3-butanediol and multi-ingredient antioxidants enhances autophagic clearance in Pompe disease Journal Articles
- Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel Journal Articles
- Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial Journal Articles
- Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial Journal Articles
- Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease. Journal Articles