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Efficacy and safety of avatrombopag in combination with immunosuppressive therapy in treatment-naïve and relapsed/refractory severe aplastic anaemia: protocol for the DIAAMOND-Ava-FIRST and DIAAMOND-Ava-NEXT Bayesian Optimal Phase II trials.
McQuilten Z, Heritier S, Fox L, Fox V, Young L, Blombery P, Cunningham I, Curnow J, Higgins A, Hiwase DK, Filshie R, Firkin F, Lacaze P, Mason K, Mills AK, Pepperell D, Patil S, Stevenson W, Szer J, Waters N, Wilson K, Ting S, Wood E. McQuilten Z, et al. Among authors: young l. BMJ Open. 2024 Jan 18;14(1):e076246. doi: 10.1136/bmjopen-2023-076246. BMJ Open. 2024. PMID: 38238183 Free PMC article.
High-efficiency base editing in the retina in primates and human tissues.
Muller A, Sullivan J, Schwarzer W, Wang M, Park-Windhol C, Hasler PW, Janeschitz-Kriegl L, Duman M, Klingler B, Matsell J, Hostettler SM, Galliker P, Hou Y, Balmer P, Virág T, Barrera LA, Young L, Xu Q, Magda DP, Kilin F, Khadka A, Moreau PH, Fellmann L, Azoulay T, Quinodoz M, Karademir D, Leppert J, Fratzl A, Kosche G, Sharma R, Montford J, Cattaneo M, Croyal M, Cronin T, Picelli S, Grison A, Cowan CS, Kusnyerik Á, Anders P, Renner M, Nagy ZZ, Szabó A, Bharti K, Rivolta C, Scholl HPN, Bryson D, Ciaramella G, Roska B, György B. Muller A, et al. Among authors: young l. Nat Med. 2025 Jan 8. doi: 10.1038/s41591-024-03422-8. Online ahead of print. Nat Med. 2025. PMID: 39779923
5,155 results