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2009 3
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Page 1
A core outcome domain set to assess cutaneous neurofibromas related to neurofibromatosis type 1 in clinical trials.
Fertitta L, Bergqvist C, Sarin KY, Plotkin SR, Moertel C, Petersen AK, Cannon A, Berman Y, Pichard DC, Röhl C, Lessing A, Brizion B, Peiffer B, Ravaud P, Tran VT, Armand ML, Moryousef S, Ferkal S, Jannic A, Ezzedine K, Wolkenstein P; Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) international collaboration. Fertitta L, et al. Br J Dermatol. 2024 Jan 23;190(2):216-225. doi: 10.1093/bjd/ljad397. Br J Dermatol. 2024. PMID: 37877514
Targeted exon skipping of NF1 exon 17 as a therapeutic for neurofibromatosis type I.
Leier A, Moore M, Liu H, Daniel M, Hyde AM, Messiaen L, Korf BR, Selvakumaran J, Ciszewski L, Lambert L, Foote J, Wallace MR, Kesterson RA, Dickson G, Popplewell L, Wallis D. Leier A, et al. Among authors: popplewell l. Mol Ther Nucleic Acids. 2022 Mar 15;28:261-278. doi: 10.1016/j.omtn.2022.03.011. eCollection 2022 Jun 14. Mol Ther Nucleic Acids. 2022. PMID: 35433111 Free PMC article.
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model.
Bourdon A, François V, Zhang L, Lafoux A, Fraysse B, Toumaniantz G, Larcher T, Girard T, Ledevin M, Lebreton C, Hivonnait A, Creismeas A, Allais M, Marie B, Guguin J, Blouin V, Remy S, Anegon I, Huchet C, Malerba A, Kao B, Le Heron A, Moullier P, Dickson G, Popplewell L, Adjali O, Montanaro F, Le Guiner C. Bourdon A, et al. Among authors: popplewell l. Gene Ther. 2022 Sep;29(9):520-535. doi: 10.1038/s41434-022-00317-6. Epub 2022 Feb 1. Gene Ther. 2022. PMID: 35105949
52 results