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Altered activation of the diaphragm in late-onset Pompe disease.
Smith BK, Corti M, Martin AD, Fuller DD, Byrne BJ. Smith BK, et al. Respir Physiol Neurobiol. 2016 Feb 1;222:11-5. doi: 10.1016/j.resp.2015.11.013. Epub 2015 Nov 28. Respir Physiol Neurobiol. 2016. PMID: 26612101 Free PMC article.
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: smith bk. 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.
281 results