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2. Adjunctive β2‐agonist treatment reduces glycogen independently of receptor‐mediated acid α‐glucosidase uptake in the limb muscles of mice with Pompe disease. Issue 5 (21st January 2014)

6. Long‐term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapy. Issue 6 (24th July 2018)

7. Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies. Issue 1 (4th February 2023)

9. Treatment outcome of twenty-two patients with guanidinoacetate methyltransferase deficiency: An international retrospective cohort study. (May 2018)