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Page 1
Pompe disease gene therapy.
Byrne BJ, Falk DJ, Pacak CA, Nayak S, Herzog RW, Elder ME, Collins SW, Conlon TJ, Clement N, Cleaver BD, Cloutier DA, Porvasnik SL, Islam S, Elmallah MK, Martin A, Smith BK, Fuller DD, Lawson LA, Mah CS. Byrne BJ, et al. Among authors: lawson la. Hum Mol Genet. 2011 Apr 15;20(R1):R61-8. doi: 10.1093/hmg/ddr174. Epub 2011 Apr 25. Hum Mol Genet. 2011. PMID: 21518733 Free PMC article. Review.
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 BK, Collins SW, Conlon TJ, Mah CS, Lawson LA, Martin AD, Fuller DD, Cleaver BD, Clément N, Phillips D, Islam S, Dobjia N, Byrne BJ. Smith BK, et al. Among authors: lawson la. Hum Gene Ther. 2013 Jun;24(6):630-40. doi: 10.1089/hum.2012.250. Hum Gene Ther. 2013. PMID: 23570273 Free PMC article. Clinical Trial.
B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease.
Elder ME, Nayak S, Collins SW, Lawson LA, Kelley JS, Herzog RW, Modica RF, Lew J, Lawrence RM, Byrne BJ. Elder ME, et al. Among authors: lawson la. J Pediatr. 2013 Sep;163(3):847-54.e1. doi: 10.1016/j.jpeds.2013.03.002. Epub 2013 Apr 16. J Pediatr. 2013. PMID: 23601496 Free PMC article. Clinical Trial.
The respiratory neuromuscular system in Pompe disease.
Fuller DD, ElMallah MK, Smith BK, Corti M, Lawson LA, Falk DJ, Byrne BJ. Fuller DD, et al. Among authors: lawson la. Respir Physiol Neurobiol. 2013 Nov 1;189(2):241-9. doi: 10.1016/j.resp.2013.06.007. Epub 2013 Jun 21. Respir Physiol Neurobiol. 2013. PMID: 23797185 Free PMC article. Review.
Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with Pompe disease.
Byrne PI, Collins S, Mah CC, Smith B, Conlon T, Martin SD, Corti M, Cleaver B, Islam S, Lawson LA. Byrne PI, et al. Among authors: lawson la. Hum Gene Ther Clin Dev. 2014 Sep;25(3):134-63. doi: 10.1089/humc.2014.2514. Hum Gene Ther Clin Dev. 2014. PMID: 25238277 Clinical Trial. No abstract available.
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 BK, Lawson LA, Tuna IS, Conlon TJ, Coleman KE, Islam S, Herzog RW, Fuller DD, Collins SW, Byrne BJ. Corti M, et al. Among authors: lawson la. Hum Gene Ther Clin Dev. 2017 Dec;28(4):208-218. doi: 10.1089/humc.2017.146. Hum Gene Ther Clin Dev. 2017. PMID: 29160099 Free PMC article. Clinical Trial.
Respiratory motor function in individuals with centronuclear myopathies.
Smith BK, Renno MS, Green MM, Sexton TM, Lawson LA, Martin AD, Corti M, Byrne BJ. Smith BK, et al. Among authors: lawson la. Muscle Nerve. 2016 Feb;53(2):214-21. doi: 10.1002/mus.24899. Epub 2015 Dec 29. Muscle Nerve. 2016. PMID: 26351754 Free PMC article.
41 results