860 results on '"Byrne, Barry J."'
Search Results
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 changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging.
5. B cell focused transient immune suppression protocol for efficient AAV readministration to the liver
6. Perspectives of the Friedreich ataxia community on gene therapy clinical trials
7. Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies
8. Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy
9. Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation
10. Current avenues of gene therapy in Pompe disease
11. Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy
12. Optogenetic activation of the tongue in spontaneously breathing mice
13. Longitudinal Observational Study of Cardiac Outcome Risk Factor Prediction in Children, Adolescents, and Adults with Barth Syndrome
14. In memoriam: Kenneth I. Berns, MD, PhD (1938–2024)
15. Kenneth I. Berns, MD, PhD [1938–2024]
16. Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency
17. Innate Immune Sensing of Adeno-Associated Virus Vectors.
18. Phenotypic implications of pathogenic variant types in Pompe disease
19. 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
20. A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease
21. Switching treatment to cipaglucosidase alfa plus miglustat positively affects motor function and quality of life in patients with late-onset Pompe disease
22. Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL
23. A plain language summary of AAV-based gene therapy: challenges and potential solutions for people with antibodies against AAV
24. 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)
25. Evolving Horizons: Adenovirus Vectors’ Timeless Influence on Cancer, Gene Therapy and Vaccines
26. Myocardial glucose and fatty acid metabolism is altered and associated with lower cardiac function in young adults with Barth syndrome
27. Gene delivery to the hypoglossal motor system: preclinical studies and translational potential
28. Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors
29. Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies.
30. The multifaceted roles of the brain glycogen.
31. Clinical Gene Therapy Trials for Pompe Disease
32. Preclinical Gene Therapy Studies for Metabolic Myopathy
33. Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies
34. 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
35. Perspectives on Best Practices for Gene Therapy Programs
36. Reduced Muscle Strength in Barth Syndrome May Be Improved by Resistance Exercise Training: A Pilot Study
37. Elevated liver glycogenolysis mediates higher blood glucose during acute exercise in Barth syndrome
38. The multifaceted roles of the brain glycogen
39. Diaphragm pacing and independent breathing in individuals with severe Pompe disease
40. Endurance Exercise Training in Young Adults with Barth Syndrome: A Pilot Study
41. Cough Effectiveness and Pulmonary Hygiene Practices in Patients with Pompe Disease
42. Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9
43. Meeting Report: 2023 Muscular Dystrophy Association Summit on ‘Safety and Challenges in Gene Therapy of Neuromuscular Diseases’
44. Dynamic respiratory muscle function in late-onset Pompe disease
45. Sustained Secretion of Human Alpha-1-Antitrypsin from Murine Muscle Transduced with Adeno-Associated Virus Vectors
46. An injectable capillary-like microstructured alginate hydrogel improves left ventricular function after myocardial infarction in rats
47. Generation and characterization of anti-Adeno-associated virus serotype 8 (AAV8) and anti-AAV9 monoclonal antibodies
48. A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice
49. Meeting Report: 2022 Muscular Dystrophy Association Summit on ‘Safety and Challenges in Gene Transfer Therapy’
50. Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory patients with Pompe disease: An open-label phase I/II study (ATB200-02)
Catalog
Books, media, physical & digital resources
Discovery Service for Jio Institute Digital Library
For full access to our library's resources, please sign in.