17 results on '"Byrne, Barry J."'
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2. Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)
3. The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy
4. Longitudinal Observational Study of Cardiac Outcome Risk Factor Prediction in Children, Adolescents, and Adults with Barth Syndrome
5. Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging
6. Phenotypic implications of pathogenic variant types in Pompe disease
7. Gene delivery to the hypoglossal motor system: preclinical studies and translational potential
8. Myocardial glucose and fatty acid metabolism is altered and associated with lower cardiac function in young adults with Barth syndrome
9. Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start
10. Cough Effectiveness and Pulmonary Hygiene Practices in Patients with Pompe Disease
11. Dynamic respiratory muscle function in late-onset Pompe disease
12. A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice
13. Endurance training ameliorates complex 3 deficiency in a mouse model of Barth syndrome
14. Substrate metabolism during basal and hyperinsulinemic conditions in adolescents and young-adults with Barth syndrome
15. Gene Delivery to Intestinal Epithelial Cells In vitro and In vivo with Recombinant Adeno-Associated Virus Types 1, 2 and 5
16. Non-invasive analysis of myoblast transplants in rodent cardiac muscle
17. Virus-Based Gene Delivery Systems
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