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Phase I study of liver depot gene therapy in late-onset Pompe disease.
Smith EC, Hopkins S, Case LE, Xu M, Walters C, Dearmey S, Han SO, Spears TG, Chichester JA, Bossen EH, Hornik CP, Cohen JL, Bali D, Kishnani PS, Koeberl DD. Smith EC, et al. Among authors: case le. Mol Ther. 2023 Jul 5;31(7):1994-2004. doi: 10.1016/j.ymthe.2023.02.014. Epub 2023 Feb 18. Mol Ther. 2023. PMID: 36805083 Clinical Trial.
Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease.
Koeberl DD, Case LE, Smith EC, Walters C, Han SO, Li Y, Chen W, Hornik CP, Huffman KM, Kraus WE, Thurberg BL, Corcoran DL, Bali D, Bursac N, Kishnani PS. Koeberl DD, et al. Among authors: case le. Mol Ther. 2018 Sep 5;26(9):2304-2314. doi: 10.1016/j.ymthe.2018.06.023. Epub 2018 Jul 5. Mol Ther. 2018. PMID: 30025991 Free PMC article. Clinical Trial.
Fractures in children with Pompe disease: a potential long-term complication.
Case LE, Hanna R, Frush DP, Krishnamurthy V, DeArmey S, Mackey J, Boney A, Morgan C, Corzo D, Bouchard S, Weber TJ, Chen YT, Kishnani PS. Case LE, et al. Pediatr Radiol. 2007 May;37(5):437-45. doi: 10.1007/s00247-007-0428-y. Epub 2007 Mar 7. Pediatr Radiol. 2007. PMID: 17342521
Early cognitive development in children with infantile Pompe disease.
Spiridigliozzi GA, Heller JH, Case LE, Jones HN, Kishnani PS. Spiridigliozzi GA, et al. Among authors: case le. Mol Genet Metab. 2012 Mar;105(3):428-32. doi: 10.1016/j.ymgme.2011.10.012. Epub 2011 Oct 28. Mol Genet Metab. 2012. PMID: 22217428 Clinical Trial.
59 results