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Lipodystrophy in methylmalonic acidemia associated with elevated FGF21 and abnormal methylmalonylation.
Manoli I, Sysol JR, Head PE, Epping MW, Gavrilova O, Crocker MK, Sloan JL, Koutsoukos SA, Wang C, Ktena YP, Mendelson S, Pass AR, Zerfas PM, Hoffmann V, Vernon HJ, Fletcher LA, Reynolds JC, Tsokos MG, Stratakis CA, Voss SD, Chen KY, Brown RJ, Hamosh A, Berry GT, Chen XS, Yanovski JA, Venditti CP. Manoli I, et al. Among authors: voss sd. JCI Insight. 2024 Feb 22;9(4):e174097. doi: 10.1172/jci.insight.174097. JCI Insight. 2024. PMID: 38271099 Free PMC article.
A phase 1 trial utilizing a pharmacokinetic endpoint to determine the optimal dose of ramucirumab in children and adolescents with relapsed or refractory solid tumors, including central nervous system tumors.
Pilbeam KL, Pradhan K, Croop J, Minard CG, Liu X, Voss SD, Isikwei E, Berg SL, Reid JM, Fox E, Weigel BJ. Pilbeam KL, et al. Among authors: voss sd. Pediatr Blood Cancer. 2024 Mar;71(3):e30817. doi: 10.1002/pbc.30817. Epub 2024 Jan 8. Pediatr Blood Cancer. 2024. PMID: 38189770 Clinical Trial.
Impact of diagnostic and end-of-induction Curie scores with tandem high-dose chemotherapy and autologous transplants for metastatic high-risk neuroblastoma: A report from the Children's Oncology Group.
Streby KA, Parisi MT, Shulkin BL, LaBarre B, Bagatell R, Diller L, Grupp SA, Matthay KK, Voss SD, Yu AL, London WB, Park JR, Yanik GA, Naranjo A. Streby KA, et al. Among authors: voss sd. Pediatr Blood Cancer. 2023 Aug;70(8):e30418. doi: 10.1002/pbc.30418. Epub 2023 May 18. Pediatr Blood Cancer. 2023. PMID: 37199022 Clinical Trial.
124 results