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1. 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

3. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

4. Longitudinal effect of eteplirsen vs. historical control on ambulation in DMD

6. Translating the genomics revolution: The need for an international gene therapy consortium for monogenic diseases

9. Effect of intracoronary delivery of autologous bone marrow mononuclear cells 2 to 3 weeks following acute myocardial infarction on left ventricular function: the LateTIME randomized trial.

10. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins.

11. Dosage-response relationships for methyl methanesulfonate in Drosophila melanogaster spermatozoa: DNA methylation per nucleotide vs. sex-linked recessive lethal frequency

12. Relative Biological Effectiveness of Tritiated Water to γ Radiation for Germ Line Mutations

14. Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.

15. Differences in Pediatricians' Income by Sex Over Time.

16. Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

17. Codon-Optimized and de novo-Synthesized E-Selectin/AAV2 Dose-Response Study for Vascular Regeneration Gene Therapy.

18. Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular Atrophy.

19. Neurological glycogen storage diseases and emerging therapeutics.

20. Innate Immune Sensing of Adeno-Associated Virus Vectors.

21. Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies.

22. 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).

23. Ventilatory Assistance Before Umbilical Cord Clamping in Extremely Preterm Infants: A Randomized Clinical Trial.

24. The multifaceted roles of the brain glycogen.

25. Video-Assisted Informed Consent in a Clinical Trial of Resuscitation of Extremely Preterm Infants: Lessons Learned.

27. 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).

28. Pediatricians' Career Satisfaction and Wellbeing by Sex Before and During the COVID-19 Pandemic.

30. B cell focused transient immune suppression protocol for efficient AAV readministration to the liver.

31. The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy.

32. Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies.

33. Meeting Report: 2023 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Therapy of Neuromuscular Diseases'.

35. Perspectives of the Friedreich ataxia community on gene therapy clinical trials.

36. Evolving Horizons: Adenovirus Vectors' Timeless Influence on Cancer, Gene Therapy and Vaccines.

37. Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy.

38. Current avenues of gene therapy in Pompe disease.

39. Elevated liver glycogenolysis mediates higher blood glucose during acute exercise in Barth syndrome.

40. Diaphragm pacing and independent breathing in individuals with severe Pompe disease.

41. Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.

42. Examining Early Career Pediatrician Characteristics, Sacrifices, and Satisfaction.

44. Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy.

45. Optogenetic activation of the tongue in spontaneously breathing mice.

47. Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.

48. Meeting Report: 2022 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Transfer Therapy'.

49. Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec.

50. Addressing the implementation gap in advanced therapeutics for spinal muscular atrophy in the era of newborn screening programs.

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