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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: lindstrom k. 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.
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: lindstrom k. J Inherit Metab Dis. 2024 Mar 3. doi: 10.1002/jimd.12724. Online ahead of print. J Inherit Metab Dis. 2024. PMID: 38433424
Best practice recommendations for the management of anxiety during the pegvaliase journey.
Bjoraker KJ, Eggerding C, Ellenberg E, Hollander S, Holmes BM, Lindstrom K, McNutt M, Miller S, Northrup H, Rogers M, Rose S, Scott M, Shim S, Wardley B, Wessenberg L, Bilder DA. Bjoraker KJ, et al. Among authors: lindstrom k. Mol Genet Metab. 2024 Jan;141(1):107737. doi: 10.1016/j.ymgme.2023.107737. Epub 2023 Nov 22. Mol Genet Metab. 2024. PMID: 38043481 Free article.
477 results