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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: collins sw. 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.
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: collins sw. 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.
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: collins sw. 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.
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: collins sw. 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.
A case report of primary amebic meningoencephalitis in North Florida.
Anjum SK, Mangrola K, Fitzpatrick G, Stockdale K, Matthias L, Ali IKM, Cope JR, O'Laughlin K, Collins S, Beal SG, Saccoccio FM. Anjum SK, et al. IDCases. 2021 Jun 28;25:e01208. doi: 10.1016/j.idcr.2021.e01208. eCollection 2021. IDCases. 2021. PMID: 34307045 Free PMC article.
14 results