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Management of early treated adolescents and young adults with phenylketonuria: Development of international consensus recommendations using a modified Delphi approach.
Burton BK, Hermida Á, Bélanger-Quintana A, Bell H, Bjoraker KJ, Christ SE, Grant ML, Harding CO, Huijbregts SCJ, Longo N, McNutt MC 2nd, Nguyen-Driver MD, Santos Pessoa AL, Rocha JC, Sacharow S, Sanchez-Valle A, Sivri HS, Vockley J, Walterfang M, Whittle S, Muntau AC. Burton BK, et al. Among authors: sacharow s. Mol Genet Metab. 2022 Sep-Oct;137(1-2):114-126. doi: 10.1016/j.ymgme.2022.07.012. Epub 2022 Jul 29. Mol Genet Metab. 2022. PMID: 36027720 Free article.
Evidence- and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria.
Longo N, Dimmock D, Levy H, Viau K, Bausell H, Bilder DA, Burton B, Gross C, Northrup H, Rohr F, Sacharow S, Sanchez-Valle A, Stuy M, Thomas J, Vockley J, Zori R, Harding CO. Longo N, et al. Among authors: sacharow s. Genet Med. 2019 Aug;21(8):1851-1867. doi: 10.1038/s41436-018-0403-z. Epub 2018 Dec 14. Genet Med. 2019. PMID: 30546086 Free PMC article.
First 1.5 years of pegvaliase clinic: Experiences and outcomes.
Sacharow S, Papaleo C, Almeida K, Goodlett B, Kritzer A, Levy H, Martell L, Wessel A, Viau K. Sacharow S, et al. Mol Genet Metab Rep. 2020 May 25;24:100603. doi: 10.1016/j.ymgmr.2020.100603. eCollection 2020 Sep. Mol Genet Metab Rep. 2020. PMID: 32489881 Free PMC article.
Safety and pharmacodynamics of an engineered E. coli Nissle for the treatment of phenylketonuria: a first-in-human phase 1/2a study.
Puurunen MK, Vockley J, Searle SL, Sacharow SJ, Phillips JA 3rd, Denney WS, Goodlett BD, Wagner DA, Blankstein L, Castillo MJ, Charbonneau MR, Isabella VM, Sethuraman VV, Riese RJ, Kurtz CB, Brennan AM. Puurunen MK, et al. Among authors: sacharow sj. Nat Metab. 2021 Aug;3(8):1125-1132. doi: 10.1038/s42255-021-00430-7. Epub 2021 Jul 22. Nat Metab. 2021. PMID: 34294923 Clinical Trial.
Use of pegvaliase in the management of phenylketonuria: Case series of early experience in US clinics.
Adams D, Andersson HC, Bausell H, Crivelly K, Eggerding C, Lah M, Lilienstein J, Lindstrom K, McNutt M, Ray JW, Saavedra H, Sacharow S, Starin D, Tiffany-Amaro J, Thomas J, Vucko E, Wessenberg LB, Whitehall K. Adams D, et al. Among authors: sacharow s. Mol Genet Metab Rep. 2021 Aug 14;28:100790. doi: 10.1016/j.ymgmr.2021.100790. eCollection 2021 Sep. Mol Genet Metab Rep. 2021. PMID: 34430209 Free PMC article.
Publisher Correction: Safety and pharmacodynamics of an engineered E. coli Nissle for the treatment of phenylketonuria: a first-in-human phase 1/2a study.
Puurunen MK, Vockley J, Searle SL, Sacharow SJ, Phillips JA 3rd, Denney WS, Goodlett BD, Wagner DA, Blankstein L, Castillo MJ, Charbonneau MR, Isabella VM, Sethuraman VV, Riese RJ, Kurtz CB, Brennan AM. Puurunen MK, et al. Among authors: sacharow sj. Nat Metab. 2022 Sep;4(9):1214. doi: 10.1038/s42255-022-00635-4. Nat Metab. 2022. PMID: 35974248 No abstract available.
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