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Pegvaliase for the treatment of phenylketonuria: Final results of a long-term phase 3 clinical trial program.
Harding CO, Longo N, Northrup H, Sacharow S, Singh R, Thomas JA, Vockley J, Zori RT, Bulloch Whitehall K, Lilienstein J, Lindstrom K, Levy DG, Jones S, Burton BK. Harding CO, et al. Among authors: lilienstein j. Mol Genet Metab Rep. 2024 Apr 23;39:101084. doi: 10.1016/j.ymgmr.2024.101084. eCollection 2024 Jun. Mol Genet Metab Rep. 2024. PMID: 38694233 Free PMC article.
Long-term preservation of intellectual functioning in sapropterin-treated infants and young children with phenylketonuria: A seven-year analysis.
Waisbren S, Burton BK, Feigenbaum A, Konczal LL, Lilienstein J, McCandless SE, Rowell R, Sanchez-Valle A, Whitehall KB, Longo N. Waisbren S, et al. Among authors: lilienstein j. Mol Genet Metab. 2021 Feb;132(2):119-127. doi: 10.1016/j.ymgme.2021.01.001. Epub 2021 Jan 13. Mol Genet Metab. 2021. PMID: 33485801 Free PMC article.
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: lilienstein j. 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.
Prevalence of comorbid conditions among adult patients diagnosed with phenylketonuria.
Burton BK, Jones KB, Cederbaum S, Rohr F, Waisbren S, Irwin DE, Kim G, Lilienstein J, Alvarez I, Jurecki E, Levy H. Burton BK, et al. Among authors: lilienstein j. Mol Genet Metab. 2018 Nov;125(3):228-234. doi: 10.1016/j.ymgme.2018.09.006. Epub 2018 Sep 12. Mol Genet Metab. 2018. PMID: 30266197 Free article.
Efficacy and safety of sapropterin before and during pregnancy: Final analysis of the Kuvan® Adult Maternal Paediatric European Registry (KAMPER) maternal and Phenylketonuria Developmental Outcomes and Safety (PKUDOS) PKU-MOMs sub-registries.
Feillet F, Ficicioglu C, Lagler FB, Longo N, Muntau AC, Burlina A, Trefz FK, van Spronsen FJ, Arnoux JB, Lindstrom K, Lilienstein J, Clague GE, Rowell R, Burton BK; KAMPER and PKUDOS investigators. Feillet F, et al. Among authors: lilienstein j. J Inherit Metab Dis. 2024 Mar 3. doi: 10.1002/jimd.12724. Online ahead of print. J Inherit Metab Dis. 2024. PMID: 38433424
Is perioperative COVID-19 really associated with worse surgical outcomes? A nationwide COVIDSurg propensity-matched analysis.
Argandykov D, Dorken-Gallastegi A, El Moheb M, Gebran A, Proaño-Zamudio JA, Bokenkamp M, Renne AM, Nepogodiev D, Bhangu A, Kaafarani HMA; COVIDSurg Collaborative. Argandykov D, et al. J Trauma Acute Care Surg. 2023 Apr 1;94(4):513-524. doi: 10.1097/TA.0000000000003859. Epub 2023 Jan 19. J Trauma Acute Care Surg. 2023. PMID: 36949053 Free PMC article.
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