• 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)
• Acute ketone supplementation in the absence of muscle glycogen utilization: Insights from McArdle disease
• Dapagliflozin and Empagliflozin in Paediatric Indications: A Systematic Review
• Diaphragm weakness in late-onset Pompe disease: A complex interplay between lower motor neuron and muscle fibre degeneration
• Efficacy and safety of enzyme replacement therapy with alglucosidase alfa for the treatment of patients with infantile-onset Pompe disease: a systematic review and metanalysis
• Empagliflozin for treating neutropenia and neutrophil dysfunction in 21 infants with glycogen storage disease 1b
• Essential dextrin structure as donor substrate for 4-alpha-glucanotransferase in glycogen debranching enzyme
• Establishing how much improvement in lung function and distance walked is clinically important for adult patients with Pompe disease
• Investigation on the treatment of empagliflozin in glycogen storage disease type Ib
• Long-term personalized high-protein, high-fat diet in pediatric patients with glycogen storage disease type IIIa: Evaluation of myopathy, metabolic control, physical activity, growth, and dietary compliance
• Medium-Chain Triglyceride Oil and Dietary Intervention Improved Body Composition and Metabolic Parameters in Children with Glycogen Storage Disease Type 1 in Jordan: A Clinical Trial
• Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease
• Paediatric hepatocellular adenomas: Lessons from a systematic review of relevant literature
• Real-world outcomes from a series of patients with late onset Pompe disease who switched from alglucosidase alfa to avalglucosidase alfa
• Treatment recommendations for glycogen storage disease type IB- associated neutropenia and neutrophil dysfunction with empagliflozin: Consensus from an international workshop
• Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS()) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data