Smith, Barbara K.

Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes

Smith, B. K., Collins, S. W., Conlon, T. J., Mah, C. S., Lawson, L. A., Martin, A. D., Fuller, D. D., Cleaver, B. D., Clément, N., Phillips, D., et al. (2013) · Human Gene Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

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Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV- hGAA ) Gene Therapy in Children Affected by Pompe Disease

Corti, M., Liberati, C., Smith, B. K., Lawson, L. A., Tuna, I. S., Conlon, T. J., Coleman, K. E., Islam, S., Herzog, R. W., Fuller, D. D., et al. (2017) · Human Gene Therapy Clinical Development

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 120

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs

Mack, D. L., Poulard, K., Goddard, M. A., Latournerie, V., Snyder, J. M., Grange, R. W., Elverman, M. R., Denard, J., Veron, P., Buscara, L., et al. (2017) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 114

Pompe disease gene therapy

Byrne, B. J., Falk, D. J., Pacak, C. A., Nayak, S., Herzog, R. W., Elder, M. E., Collins, S. W., Conlon, T. J., Clement, N., Cleaver, B. D., et al. (2011) · Human Molecular Genetics

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

Cited by 91

Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy

Byrne, B. J., Fuller, D. D., Smith, B. K., Clement, N., Coleman, K., Cleaver, B., Vaught, L., Falk, D. J., McCall, A., & Corti, M. (2019) · Annals of Translational Medicine

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

Cited by 51

Altered activation of the tibialis anterior in individuals with Pompe disease: Implications for motor unit dysfunction: Neuromuscular Activation in Pompe

Corti, M., Smith, B. K., Falk, D. J., Lawson, L. A., Fuller, D. D., Subramony, S. H., Byrne, B. J., & Christou, E. A. (2015) · Muscle & Nerve

Lysosomal Storage Disorders ResearchMuscle Physiology and Disorders

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