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Journal Abstract Search

323 related items for PubMed ID: 28282402

  • 1. Formulation and PEGylation optimization of the therapeutic PEGylated phenylalanine ammonia lyase for the treatment of phenylketonuria.
    Bell SM, Wendt DJ, Zhang Y, Taylor TW, Long S, Tsuruda L, Zhao B, Laipis P, Fitzpatrick PA.
    PLoS One; 2017; 12(3):e0173269. PubMed ID: 28282402
    [Abstract] [Full Text] [Related]

  • 2. Development of pegylated forms of recombinant Rhodosporidium toruloides phenylalanine ammonia-lyase for the treatment of classical phenylketonuria.
    Gámez A, Sarkissian CN, Wang L, Kim W, Straub M, Patch MG, Chen L, Striepeke S, Fitzpatrick P, Lemontt JF, O'Neill C, Scriver CR, Stevens RC.
    Mol Ther; 2005 Jun; 11(6):986-9. PubMed ID: 15922970
    [Abstract] [Full Text] [Related]

  • 3. Erythrocyte-mediated delivery of phenylalanine ammonia lyase for the treatment of phenylketonuria in BTBR-Pah(enu2) mice.
    Rossi L, Pierigè F, Carducci C, Gabucci C, Pascucci T, Canonico B, Bell SM, Fitzpatrick PA, Leuzzi V, Magnani M.
    J Control Release; 2014 Nov 28; 194():37-44. PubMed ID: 25151978
    [Abstract] [Full Text] [Related]

  • 4. A new therapy prevents intellectual disability in mouse with phenylketonuria.
    Pascucci T, Rossi L, Colamartino M, Gabucci C, Carducci C, Valzania A, Sasso V, Bigini N, Pierigè F, Viscomi MT, Ventura R, Cabib S, Magnani M, Puglisi-Allegra S, Leuzzi V.
    Mol Genet Metab; 2018 May 28; 124(1):39-49. PubMed ID: 29661557
    [Abstract] [Full Text] [Related]

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  • 6. Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial.
    Longo N, Harding CO, Burton BK, Grange DK, Vockley J, Wasserstein M, Rice GM, Dorenbaum A, Neuenburg JK, Musson DG, Gu Z, Sile S.
    Lancet; 2014 Jul 05; 384(9937):37-44. PubMed ID: 24743000
    [Abstract] [Full Text] [Related]

  • 7. Phenylalanine ammonia lyase, enzyme substitution therapy for phenylketonuria, where are we now?
    Sarkissian CN, Gámez A.
    Mol Genet Metab; 2005 Dec 05; 86 Suppl 1():S22-6. PubMed ID: 16165390
    [Abstract] [Full Text] [Related]

  • 8. Pegvaliase for the treatment of phenylketonuria: Results of the phase 2 dose-finding studies with long-term follow-up.
    Burton BK, Longo N, Vockley J, Grange DK, Harding CO, Decker C, Li M, Lau K, Rosen O, Larimore K, Thomas J, PAL-002 and PAL-004 Investigators.
    Mol Genet Metab; 2020 Aug 05; 130(4):239-246. PubMed ID: 32593547
    [Abstract] [Full Text] [Related]

  • 9. Structure-based chemical modification strategy for enzyme replacement treatment of phenylketonuria.
    Wang L, Gamez A, Sarkissian CN, Straub M, Patch MG, Han GW, Striepeke S, Fitzpatrick P, Scriver CR, Stevens RC.
    Mol Genet Metab; 2005 Aug 05; 86(1-2):134-40. PubMed ID: 16006165
    [Abstract] [Full Text] [Related]

  • 10. Phenylalanine ammonia lyase (PAL): From discovery to enzyme substitution therapy for phenylketonuria.
    Levy HL, Sarkissian CN, Scriver CR.
    Mol Genet Metab; 2018 Aug 05; 124(4):223-229. PubMed ID: 29941359
    [Abstract] [Full Text] [Related]

  • 11. Induction, titration, and maintenance dosing regimen in a phase 2 study of pegvaliase for control of blood phenylalanine in adults with phenylketonuria.
    Zori R, Thomas JA, Shur N, Rizzo WB, Decker C, Rosen O, Li M, Schweighardt B, Larimore K, Longo N.
    Mol Genet Metab; 2018 Nov 05; 125(3):217-227. PubMed ID: 30146451
    [Abstract] [Full Text] [Related]

  • 12. Pegvaliase for the treatment of phenylketonuria: Results of a long-term phase 3 clinical trial program (PRISM).
    Thomas J, Levy H, Amato S, Vockley J, Zori R, Dimmock D, Harding CO, Bilder DA, Weng HH, Olbertz J, Merilainen M, Jiang J, Larimore K, Gupta S, Gu Z, Northrup H, PRISM investigators.
    Mol Genet Metab; 2018 May 05; 124(1):27-38. PubMed ID: 29653686
    [Abstract] [Full Text] [Related]

  • 13. Phenylalanine ammonia-lyase modified with polyethylene glycol: potential therapeutic agent for phenylketonuria.
    Ikeda K, Schiltz E, Fujii T, Takahashi M, Mitsui K, Kodera Y, Matsushima A, Inada Y, Schulz GE, Nishimura H.
    Amino Acids; 2005 Nov 05; 29(3):283-7. PubMed ID: 15977040
    [Abstract] [Full Text] [Related]

  • 14. Alternative therapies to address the unmet medical needs of patients with phenylketonuria.
    Blau N, Longo N.
    Expert Opin Pharmacother; 2015 Apr 05; 16(6):791-800. PubMed ID: 25660215
    [Abstract] [Full Text] [Related]

  • 15. Evaluation of orally administered PEGylated phenylalanine ammonia lyase in mice for the treatment of Phenylketonuria.
    Sarkissian CN, Kang TS, Gámez A, Scriver CR, Stevens RC.
    Mol Genet Metab; 2011 Nov 05; 104(3):249-54. PubMed ID: 21803624
    [Abstract] [Full Text] [Related]

  • 16. Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study.
    Longo N, Zori R, Wasserstein MP, Vockley J, Burton BK, Decker C, Li M, Lau K, Jiang J, Larimore K, Thomas JA.
    Orphanet J Rare Dis; 2018 Jul 04; 13(1):108. PubMed ID: 29973227
    [Abstract] [Full Text] [Related]

  • 17. Partial rescue of neuropathology in the murine model of PKU following administration of recombinant phenylalanine ammonia lyase (pegvaliase).
    Goldfinger M, Zeile WL, Corado CR, O'Neill CA, Tsuruda LS, Laipis PJ, Cooper JD.
    Mol Genet Metab; 2017 Sep 04; 122(1-2):33-35. PubMed ID: 28506393
    [Abstract] [Full Text] [Related]

  • 18. Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial.
    Harding CO, Amato RS, Stuy M, Longo N, Burton BK, Posner J, Weng HH, Merilainen M, Gu Z, Jiang J, Vockley J, PRISM-2 Investigators.
    Mol Genet Metab; 2018 May 04; 124(1):20-26. PubMed ID: 29628378
    [Abstract] [Full Text] [Related]

  • 19. Trends in enzyme therapy for phenylketonuria.
    Kim W, Erlandsen H, Surendran S, Stevens RC, Gamez A, Michols-Matalon K, Tyring SK, Matalon R.
    Mol Ther; 2004 Aug 04; 10(2):220-4. PubMed ID: 15294168
    [Abstract] [Full Text] [Related]

  • 20. Toward PKU enzyme replacement therapy: PEGylation with activity retention for three forms of recombinant phenylalanine hydroxylase.
    Gámez A, Wang L, Straub M, Patch MG, Stevens RC.
    Mol Ther; 2004 Jan 04; 9(1):124-9. PubMed ID: 14741785
    [Abstract] [Full Text] [Related]

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