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Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy.
Mayhew AG, Moat D, McDermott MP, Eagle M, Griggs RC, King WM, James MK, Muni-Lofra R, Shillington A, Gregson S, Pallant L, Skura C, Staudt LA, Eichinger K, McMurchie H, Rabb R, Di Marco M, Brown S, Zanin R, Arnoldi MT, McIntyre M, Wilson A, Alfano LN, Lowes LP, Blomgren C, Milev E, Iodice M, Pasternak A, Chiu A, Lehnert I, Claus N, Dieruf KA, Rolle E, Nicorici A, Andres B, Hobbiebrunken E, Roetmann G, Kern V, Civitello M, Vogt S, Hayes MJ, Scholtes C, Lacroix C, Gunn T, Warner S, Newman J, Barp A, Kundrat K, Kovelman S, Powers PJ, Guglieri M; Muscle Study Group and TREAT-NMD. Mayhew AG, et al. Among authors: brown s. Neuromuscul Disord. 2022 Jun;32(6):460-467. doi: 10.1016/j.nmd.2022.02.012. Epub 2022 Feb 26. Neuromuscul Disord. 2022. PMID: 35618576 Free article.
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators. Mah JK, et al. JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178. JAMA Netw Open. 2022. PMID: 35076703 Free PMC article. Clinical Trial.
The role of family history in predicting germline pathogenic variant carriers who develop pancreatic cancer: Results of a multicenter collaboration.
Karloski E, Dudley B, Diergaarde B, Blanco A, Everett JN, Levinson E, Rangarajan T, Stanich PP, Childers K, Brown S, Drogan C, Cavestro GM, Gordon K, Singh A, Simeone DM, Reich H, Kastrinos F, Zakalik D, Hampel H, Pearlman R, Gordon OK, Kupfer SS, Puzzono M, Zuppardo RA, Brand RE. Karloski E, et al. Among authors: brown s. Cancer. 2024 May 29. doi: 10.1002/cncr.35383. Online ahead of print. Cancer. 2024. PMID: 38809542
Variation in structural motifs within SARS-related coronavirus spike proteins.
Hills FR, Eruera AR, Hodgkinson-Bean J, Jorge F, Easingwood R, Brown SHJ, Bouwer JC, Li YP, Burga LN, Bostina M. Hills FR, et al. Among authors: brown shj. PLoS Pathog. 2024 May 28;20(5):e1012158. doi: 10.1371/journal.ppat.1012158. Online ahead of print. PLoS Pathog. 2024. PMID: 38805567 Free article.
AAV-mediated gene therapy for Sialidosis.
van de Vlekkert D, Hu H, Weesner JA, Fremuth LE, Brown SA, Lu M, Gomero E, Campos Y, Sheppard H, Alessandra d'Azzo. van de Vlekkert D, et al. Among authors: brown sa. Mol Ther. 2024 May 24:S1525-0016(24)00331-9. doi: 10.1016/j.ymthe.2024.05.029. Online ahead of print. Mol Ther. 2024. PMID: 38796704
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