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51. Nicholas Muzyczka, PhD

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

62. Correction to: Reduced Muscle Strength in Barth Syndrome May Be Improved by Resistance Exercise Training: A Pilot Study

65. Additional file 4 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

66. Additional file 2 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

67. Additional file 6 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

68. Additional file 3 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

69. Additional file 5 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

70. Additional file 1 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

81. Human Gene Therapy for RPE65 Isomerase Deficiency Activates the Retinoid Cycle of Vision but with Slow Rod Kinetics

82. Diaphragm pacing as a rehabilitative tool for patients with pompe disease who are ventilator-dependent: case series

83. 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

85. Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort

88. Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort

89. Resistance exercise training with protein supplementation improves skeletal muscle strength and improves quality of life in late adolescents and young adults with Barth syndrome: A pilot study

100. Current Clinical Applications of In Vivo Gene Therapy with AAVs

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