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Long-term neuropsychologic outcome of pre-emptive mTOR inhibitor treatment in children with tuberous sclerosis complex (TSC) under 4 months of age (PROTECT), a two-arm, randomized, observer-blind, controlled phase IIb national multicentre clinical trial: study protocol.
Driedger JH, Schröter J; PROTECT-Study Group; Syrbe S, Saffari A. Driedger JH, et al. Orphanet J Rare Dis. 2025 Jan 6;20(1):2. doi: 10.1186/s13023-024-03495-1. Orphanet J Rare Dis. 2025. PMID: 39762914 Free PMC article.
Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study.
Weiß C, Becker LL, Friese J, Blaschek A, Hahn A, Illsinger S, Schwartz O, Bernert G, Hagen MV, Husain RA, Goldhahn K, Kirschner J, Pechmann A, Flotats-Bastardas M, Schreiber G, Schara U, Plecko B, Trollmann R, Horber V, Wilichowski E, Baumann M, Klein A, Eisenkölbl A, Köhler C, Stettner GM, Cirak S, Hasselmann O, Kaindl AM, Garbade SF, Johannsen J, Ziegler A; SMArtCARE and Swiss-Reg-NMD study group. Weiß C, et al. Lancet Reg Health Eur. 2024 Oct 7;47:101092. doi: 10.1016/j.lanepe.2024.101092. eCollection 2024 Dec. Lancet Reg Health Eur. 2024. PMID: 39434961 Free PMC article.
Azooxanthellate Palythoa (Cnidaria: Anthozoa) Genomes Reveal Toxin-related Gene Clusters and Loss of Neuronal Genes in Hexacorals.
Yoshioka Y, Yamashita H, Uchida T, Shinzato C, Kawamitsu M, Fourreau CJL, Castelló GM, Fiedler BK, van den Eeckhout TM, Borghi S, Reimer JD, Shoguchi E. Yoshioka Y, et al. Among authors: fiedler bk. Genome Biol Evol. 2024 Sep 3;16(9):evae197. doi: 10.1093/gbe/evae197. Genome Biol Evol. 2024. PMID: 39240721 Free PMC article.
RIPK3 and caspase-8 interpret cytokine signals to regulate ILC3 survival in the gut.
Joseph AM, Ahmed A, Goc J, Horn V, Fiedler B, Garone D, Grigg JB, Uddin J, Teng F, Fritsch M, Vivier E, Sonnenberg GF. Joseph AM, et al. Among authors: fiedler b. Mucosal Immunol. 2024 Dec;17(6):1212-1221. doi: 10.1016/j.mucimm.2024.08.004. Epub 2024 Aug 11. Mucosal Immunol. 2024. PMID: 39137882 Free article.
Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled Trial.
Schwartz O, Vill K, Pfaffenlehner M, Behrens M, Weiß C, Johannsen J, Friese J, Hahn A, Ziegler A, Illsinger S, Smitka M, von Moers A, Kölbel H, Schreiber G, Kaiser N, Wilichowski E, Flotats-Bastardas M, Husain RA, Baumann M, Köhler C, Trollmann R, Schwerin-Nagel A, Eisenkölbl A, Schimmel M, Fleger M, Kauffmann B, Wiegand G, Baumgartner M, Rauscher C, Cirak S, Gläser D, Bernert G, Hagenacker T, Goldbach S, Probst-Schendzielorz K, Lochmüller H, Müller-Felber W, Schara-Schmidt U, Walter MC, Kirschner J, Pechmann A; SMARTCARE study group. Schwartz O, et al. JAMA Pediatr. 2024 Jun 1;178(6):540-547. doi: 10.1001/jamapediatrics.2024.0492. JAMA Pediatr. 2024. PMID: 38587854 Clinical Trial.
166 results