Scientific Publications

The following list scientific articles and research papers regarding Sodium Phenylbuterate, published since 2005. For articles published prior to 2005 please inquire by email to info@ampolgen.com 

Cancer Research

Cancer Treatment

Bladder Cancer

1.    Mortazavi, A; Hoot, DR; Carlton, PS; Wang, S; Degroff, VL.; Lu, Q; Kulp, S; Chen, C-S; Clinton, SK.  Inhibition of cell growth and induction of apoptosis in bladder cancer cell lines by a novel histone deacctylase inhibitor derived from phenylbutyrate.  Proceedings of the American Association for Cancer Research Annual Meeting 2005;46(Supplement S):422.

2.    Picard, V; Bergeron, A; Larue, H; Fradet, Y.  MAGE-A9 mRNA and protein expression in bladder cancer.  Int J Cancer 2007;120(10):2170-2177.

Brain Tumors

1.    Phuphanich, S; Baker, SD; Grossman, SA; Carson, KA; Gilbert, MR; Fisher, JD; Carducci, MA.  Oral sodium phenylbutyrate in patients with recurrent malignant gliomas: a dose escalation and pharmacologic study.  Neuro-oncol 2005;7(2):177-182.

2.    Vila-Carriles, WH; Kovacs, GG; Jovov, B; Zhou, Z-H; Pahwa, AK; Colby, G; Esimai, O; Gillespie, GY; Mapstone, TB; Markert, JM; Fuller, CM; Bubien, JK; Benos, DJ.  Surface expression of ASIC2 inhibits the amiloride-sensitive current and migration of glioma cells.  J Biol Chem 2006;281(28):19220-19232.

3.    Vila-Carriles, WH; Kovacs, GG; Bubien, JK; Gillespie, GY; Fuller, CM; Benos, DJ.  Cellular localization of acid sensing ion channels in human astrocytes and gliomas.  FASEB Journal 2006;20(4, Part 1):A325.

4.    Vila-Carriles, WH; Zhou, Z-H; Bubien, JK; Fuller, CM; Benos, DJ.  Participation of the chaperone Hsc70 in the trafficking and functional expression of ASIC2 in glioma cells.  J Biol Chem 2007;282(47):34381-34391.

5.    Svechnikova, I; Almqvist, PM; Ekstroem, TJ.  HDAC inhibitors effectively induce cell type-specific differentiation in human glioblastoma cell lines of different origin.  Int J Oncol 2008;32(4):821-827.

6.    Entin-Meer, M; Rephaeli, A; Yang, X; Nudelman, A; Nudelman, A; Haas-Kogan, DA.  AN-113, a novel prodrug of 4-phenylbutyrate with increased anti-neoplastic activity in glioma cell lines.  Cancer Lett 2007;253(2):205-214.

Breast Cancer

1.    Christov, K; Grubbs, C; Lubet, R.  Altered cell proliferation and apoptosis as biomarkers for identifying preventive/therapeutic agents against chemically induced mammary cancers.  Breast Cancer Research and Treatment 2006;100(Suppl. 1):S58.

2.    Christov, Konstantin; Grubbs, Clinton J; Shilkaitis, Anne; Juliana, M Margaret; Lubet, Ronald A.  Short-term modulation of cell proliferation and apoptosis and preventive/therapeutic efficacy of various agents in a mammary cancer model.  Clin Cancer Res 2007;13(18, Part 1):5488-5496.

3.    Christov, K; Grubbs, C; Juliana, M; Luber, R.  Correlation of the preventive/therapeutic efficacy of agents in the methylnitrosourea (MNU) mammary cancer model with changes in cell proliferation and apoptosis following short-term treatment.  Proceedings of the American Association for Cancer Research Annual Meeting 2007;48:997.

Colon Cancer

1.    Lea, MA; Ibeh, C; Shah, N; Moyer, MP.  Enhanced differentiation of colon cancer cells induced by combinations of inhibitors of kinases and of historic deacerylases.  Proceedings of the American Association for Cancer Research Annual Meeting 2006;47:1184.

2.    Sung, MW; Waxman, S.  Combination of cytotoxic-differentiation therapy with 5-fluorouracil and phenylbutyrate in patients with advanced colorectal cancer.  Anticancer Res 2007;27(2):995-1001.

Combination Treatment

1.    Hao, C-l; Tang, K-j; Chen, S; Xing, H-y; Wang, M; Wang, J-x.  5-Aza-2'-deoxycytidine enhances differentiation and apoptosis induced by phenylbutyrate in Kasumi-1 cells.  Zhonghua Zhong Liu Za Zhi 2005;27(3):148-151.

2.    Rudek, MA; Zhao, M; He, P; Hartke, C; Gilbert, J; Gore, SD; Carducci, MA; Baker, SD.  Pharmacokinetics of 5-azacitidine administered with phenylbutyrate in patients with refractory solid tumors or hematologic malignancies.  J Clin Oncol 2005;23(17):3906-3911.

3.    Gore, SD.; Jiemjit, A; Silverman, LB.; Aucott, T; Baylin, S; Carraway, H; Douses, T; Fandy, T; Herman, J; Karp, JE.; Licht, JD.; Murgo, AJ.; Odchimar-Reissig, R; Smith, BD; Zwiebel, JA.; Sugar, E.  Combined Methyltransferase/Histone deacetylase inhibition with 5Azacitidine and MS-275 in patients with MDS, CMMoL and AML: Clinical response, Histone Acetylation and DNA damage.  Blood 2006;108(11, part 1):156A-157A.

4.    Schniewind, B; Heintz, K; Kurdow, R; Ammerpohl, O; Trauzold, A; Emme, D; Dohrmann, P; Kalthoff, H.  Combination phenylbutyrate/gemcitabine therapy effectively inhibits in vitro and in vivo growth of NSCLC by intrinsic apoptotic pathways.  J Carcinog 2006;5:25.

5.    Gore, SD; Baylin, S; Sugar, E; Carraway, H; Miller, CB; Carducci, M; Grever, M; Galm, O; Dauses, T; Karp, JE; Rudek, MA; Zhao, M; Smith, BD; Manning, J; Jiemjit, A; Dover, G; Mays, A; Zwiebel, J; Murgo, A; Weng, L-J; Herman, JG.  Combined DNA methyltransferase and histone deacetylase inhibition in the treatment of myeloid neoplasms.  Cancer Res 2006;66(12):6361-6369.

6.    Carraway, HE; Gore, SD.  Addition of histone deacetylase inhibitors in combination therapy.  J Clin Oncol 2007;25(15):1955-1956.

7.    Verheul, HMW; Qian, DZ; Carducci, MA; Pili, R.  Sequence-dependent antitumor effects of differentiation agents in combination with cell cycle-dependent cytotoxic drugs.  Cancer Chemother Pharmacol 2007;60(3):329-339.

8.    Ammerpohl, O; Trauzold, A; Schniewind, B; Griep, U; Pilarsky, C; Grutzmann, R; Saeger, H-D; Janssen, O; Sipos, B; Kloppel, G; Kalthoff, H.  Complementary effects of HDAC inhibitor 4-PB on gap junction communication and cellular export mechanisms support restoration of chemosensitivity of PDAC cells.  Br J Cancer 2007;96(1):73-81.

9.    Perron, ME; Plourde, F; Guerard, S; Huynh, L; Leroux, JC; et al.  An investigation on the use of tributyrin nanoemulsions for docetaxel delivery.  J Drug Deliv Sci Technol 2008;18:189-195.

10.Su, J; Zhang, NN; Ho, PC.  Evaluation of the pharmacokinetics of all-trans-retinoic acid (ATRA) in Wistar rats after intravenous administration of ATRA loaded into tributyrin submicron emulsion and its cellular activity on Caco-2 and HepG2 cell lines.  J Pharm Sci 2008;97:2844-2853.

11.Huynh, L; Grant, J; Leroux, JC; Delmas, P; Allen, C.  Predicting the solubility of the anti-cancer agent docetaxel in small molecule excipients using computational methods.  Pharm Res 2008;25:147-157.

12.Jiemjit, A; Fandy, TE; Baylin, SB; Carraway, H; Herman, JG; Gore, SD.  Sequential administration of DNA methyltransferase inhibitors (DNMTi) and histone deacetylase inhibitors (HDACi) induces apoptosis with caspase and reactive oxygen species (ROS)-dependent synergy.  Proceedings of the American Association for Cancer Research Annual Meeting 2007;48:249.

 

Esophageal Cancer

1.    Brock, MV.  Clinical aspects of molecular biology for the diagnosis and treatment of esophageal cancer.  Esophagus 2006;3(3):91-94.

2.    Beyer, SJ; Kulp, SK; Baird, M; Auer, H; Kornacker, K; Chen, C-S; Beer, DG; Kresty, LA.  The effects of a phenylbutyrate-derived histone deacetylase inhibitor (HDAC-42) on acid-induced gene expression patterns of SEG-1 human esophageal adenocarcinoma cells.  Proceedings of the American Association for Cancer Research Annual Meeting 2006;47:1128-1129.

Ewing Sarcoma

1.    Hurtubise, A; Bernstein, ML; Momparler, RL.  Preclinical evaluation of the antineoplastic action of 5-aza-2'-deoxycytidine and different histone deacetylase inhibitors on human Ewing's sarcoma cells.  Cancer Cell Int 2008;8:16.

General

1.    Garcia-Manero, G; Issa, J-P.  Histone deacetylase inhibitors: A review of their clinical status as antineoplastic agents.  Cancer Investigation 2005;23(7):635-642.

2.    Mack, GS.  Epigenetic cancer therapy makes headway.  Journal of the National Cancer Institute 2006;98(20):1443-1444.

3.    Fouladi, M.  Histone deacetylase inhibitors in cancer therapy.  Cancer Investigation 2006;24(5):521-527.

4.    Galm, O; Herman, JG; Baylin, SB.  The fundamental role of epigenetics in hematopoietic malignancies.  Blood Rev 2006;20(1):1-13.

5.    Lubbert, M.; Claus, R.  Epigenetic inactivation of gene expression. New therapeutic targets in hematooncology.  Onkologe 2007;13(1):46-55.

6.    Cortez, CC; Jones, PA.  Chromatin, cancer and drug therapies.  Mutat Res - Fundam Mol Mech Mutagen 2008;647(1-2):44-51.

7.    Monneret, C.  Histone deacetylase inhibitors. Eur J Med Chem 2005;40(1):1-13.

8.    Holm, E; Schade, I.  Effects of short-chain and saturated long-chain fatty acids on tumor growth - in vitro and experimental in vivo effects.  Aktuelle Ernahrungsmedizin 2008;33(5):225-230.

 

 

Head & Neck Cancer

1.    Gao J; Luan X-y; Xu F-l; Liu D-y; Lei D-p.  Effects of sodium phenylbutyrate alone or in combination with fluorouracil or cisplatin on laryngeal carcinoma Hep-2 cell line.  Zhongguo Xinyao yu Linchuang Zazhi 2005;24(10):778-781.

2.    Gao, J; Ruan, X; Pan, X; Xu, F; Lei, D; Liu, D.  The effect of sodium phenylbutyrate to agents used in induction chemotherapy on laryngeal carcinoma cells Hep-2 in vitro.  Lin Chuang Er Bi Yan Hou Ke Za Zhi 2005;19(15):680-682.

3.    Hattori, Y; Fukushima, M; Maitani, Y.  Non-viral delivery of the connexin 43 gene with histone deacetylase inhibitor to human nasopharyngeal tumor cells enhances gene expression and inhibits in vivo tumor growth.  Int J Oncol 2007; 30(6):1427-1439.

4.    Burkitt, K; Ljungman, M.  Phenylbutyrate interferes with the Fanconi anemia and BRCA pathway and sensitizes head and neck cancer cells to cisplatin.  Mol Cancer 2008;7:24.

Leukemia

1.    Sun, J; Liu, S; Yu, J; Wei, M; Mao, C; Ding, H; Kearney, J; Huynh, L; Paschka, P; Wang, D; Klisovic, RB.; Perrotti, D; Chen, C-S; Blum, WG; Marcucci, G.  Characterization of HDACI OSU42 as a novel histone deacetylase inhibitor in AML cell lines.  Blood 2006;108(11 part 1):563A.

2.    Luebbert, M.  Combined targeting of the epigenetic silence in leukemia: Cooperating activities of DNA methylation and histone deacetylation inhibitors.  Leukemia Research 2005;29(7):727-728.

3.    Maslak, P; Chanel, S; Camacho, LH; Soignet, S; Pandolfi, PP; Guernah, I; Warrell, R; Nimer, S.  Pilot study of combination transcriptional modulation therapy with sodium phenylbutyrate and 5-azacytidine in patients with acute myeloid leukemia or myelodysplastic syndrome.  Leukemia 2006;20(2):212-217.

4.    West, DA; Lucas, DM; Davis, ME; De lay, MD; Johnson, AJ; Guster, SE; Freitas, MA; Parthun, MR; Wang, D; Kulp, SK; Grever, MR; Chen, C-S; Byrd, JC.  The novel histone deacetylase inhibitor OSU-HDAC42 has class I and II histone deacetylase (HDAC) inhibitory activity and represents a novel therapy for chronic lymphocytic leukemia.  Blood 2006;108(11, Part 1):794A-795A.

5.    Cunha De Santis, G; de Barros Tamarozzi, M; Sousa, RB; Moreno, SE; Secco, D; Garcia, AB; Lima, ASG; Faccioli, LH; Falcoao, RP; Cunha, FQ; Rego, EM.  Adhesion molecules and Differentiation Syndrome: phenotypic and functional analysis of the effect of ATRA, As2O3, phenylbutyrate, and G-CSF in acute promyelocytic leukemia.  Haematologica 2007;92(12):1615-1622.

6.    Koutsourea, AI; Fousteris, MA; Arsenou, ES; Papageorgiou, A; Pairas, GN; Nikolaropoulos, SS.  Rational design, synthesis, and in vivo evaluation of the antileukemic activity of six new alkylating steroidal esters.  Bioorg Med Chem 2008;16(9):5207-5215.

7.    Estey, E.  New drugs in acute myeloid leukemia.  Seminars in Oncology 2008;35(4):439-448.

Liver Cancer

1.    Meng, M; Wang, C-T; Jiang, J-M; Zhang, J-C; Jiang, J-J; Jin, C-J.  Expression of histone deacetyase 4 in human liver carcinoma cell line Bel-7402 and its significance.  Chinese Journal of Cancer Biotherapy Chin. J. Cancer Biother 2007;14(2):153-157.

2.    Lu, Y-S; Kashida, Y; Kulp, SK; Wang, Y-C; Wang, D; Hung, J-H; Tang, M; Lin, Z-Z; Chen, T-J; Cheng, A-L; Chen, C-S.  Efficacy of a novel histone deacetylase inhibitor in murine models of hepatocellular carcinoma.  Hepatology 2007;46(4):1119-1130.

3.    Svechnikova, I; Ammerpohl, O; Ekstroem, TJ.  p21waf1/Cip1 partially mediates apoptosis in hepatocellular carcinoma cells.  Biochem Biophys Res Commun 2007;354(2):466-471.

4.    Lu, Y-S; Kashida, Y; Hung, J-H; Chen, C-S; Wang, Y-C; Cheng, A-L; Chen, C-S.  In vivo efficacy of OSU-HDAC42, a novel phenylbutyrate-based histone deacetylase inhibitor, in human hepatocellular carcinoma animal models.  Proceedings of the American Association for Cancer Research Annual Meeting 2007;48:169.

5.    Wang, H-E; Wu, H-C; Kao, S-J; Tseng, F-W; Wang, Y-S; Yu, H-M; Chou, S-L; Yen, S-H; Chi, K-H.  Modulation of 5-fluorouracil cytotoxicity through thymidylate synthase and NF-kappaB down-regulation and its application on the radiolabelled iododeoxyuridine therapy on human hepatoma cell.  Biochem Pharmacol 2005;69(4):617-626.

6.    Meng, M; Jiang, JM.; Liu, H; In, CY; Zhu, JR.  Effects of sodium phenylbutyrate on differentiation and induction of the P<sup>21WAF1/CIP1</sup> anti-oncogene in human liver carcinoma cell lines.  Chin J Dig Dis 2005;6(4):189-192.

Lung Cancer

1.    Lyon, CM; Klinge, D; Liechty, KC; Belinsky, SA.  DNA demethylating agents and a PPA-gamma agonist cooperate to induce apoptosis in lung cancer cell lines.  Proceedings of the American Association for Cancer Research Annual Meeting 2006;47:10.

2.    Lyon, CM; Klinge, D; Liechty, KC; Grimes, MJ; Grimes, J; Thomas, C; March, T; Stidley, C; Keith, R; Belinsky, SA.  Rosiglitazone prevents the progression of pre-invasive lung cancer in a murine model.  Proceedings of the American Association for Cancer Research Annual Meeting 2007;48:395.

Myelodysplastic Syndrome

1.    Meletis, J; Viniou, N; Terpos, E.  Novel agents for the management of myelodysplastic syndromes.  Med Sci Monit 2006;12(9):RA194-RA206.

2.    Claus, R; Router, B; Loubbert, M.  Targets of epigenetic therapy - Gene reactivation as a novel approach in MDS treatment.  Cancer Treat Rev 2007;33(Suppl. 1):S47-S52.

3.    Luebbert, M.  Which myelodysplastic syndromes patients are candidates for treatment with decitabine.  Leukemia Research 2007;31(Suppl. 1):S20.

4.    Gore SD.  Do histone deacetylase inhibitors have a place in the treatment of myelodysplastic syndromes?  Leukemia Research 2007;31(Suppl. 1):S21.

Non-Hodgkin’s Lymphoma

1.    Phillips, JA; Griffin, BE.  Pilot study of sodium phenylbutyrate as adjuvant in cyclophosphamide-resistant endemic Burkitt's lymphoma.  Trans R Soc Trop Med Hyg 2007;101(12):1265-1269.

Osteosarcoma

1.    Murahari, S; Jalkanen, AL.; Kulp, SK.; Chen, C-S; Jubala, CM; Fosmire, SP; Modiano, JF; Fossey, SL; London, CA; Kisseberth, WC.  OSU-HDAC42, a novel histone deacetylase inhibitor with potent antitumor effects on human and canine osteosarcoma cells.  Proceedings of the American Association for Cancer Research Annual Meeting 2008;49:578.

Prostate Cancer

1.    Milkevitch, M; Shim, H; Pilatus, U; Pickup, S; Wehrle, JP; Samid, D; Poptani, H; Glickson, JD; Delikatny, EJ.  Increases in NMR-visible lipid and glycerophosphocholine during phenylbutyrate-induced apoptosis in human prostate cancer cells. Biochim Biophys Acta 2005; 1734(1):1-12.

2.    Kulp, SK; Chen, C-S; Wang, D-S; Chen, C-Y; Chen, C-S.  Antitumor effects of a novel phenylbutyrate-based histone deacetylase inhibitor, (S)-HDAC-42, in prostate cancer.  Clin Cancer Res 2006;12(17):5199-5206.

3.    Milkevitch, M; Jeitner, TM; Beardsley, NJ; Delikatny, EJ.  Lovastatin enhances phenylbutyrate-induced MR-visible glycerophosphocholine but not apoptosis in DU145 prostate cells.  Biochim Biophys Acta 2007;1771(9):1166-1176.

Cardioprotection

1.    Daosukho, C; Chen, Y; Noel, T; Sompol, P; Nithipongvanitch, R; Velez, JM; Oberley, TD; St Clair, DK.  Phenylbutyrate, a histone deacetylase inhibitor, protects against Adriamycin-induced cardiac injury.  Free Radic Biol Med 2007;42(12):1818-1825.

Phase I Study

1.    Camacho, LH; Olson, J; Tong, WP; Young, CW; Spriggs, DR; Malkin, MG.  Phase I dose escalation clinical trial of phenylbutyrate sodium administered twice daily to patients with advanced solid tumors.  Invest New Drugs 2007;25(2):131-138.

Phenylbutyrate Derivatives

1.    Sampathkumar, S.-G; Jones, MB.; Meledeo, MA; Hida, K; Sheh, T; Gomatputra, P; Yarema, KJ.  Carbohydrate-based small molecules as anti-cancer drugs: Short chain fatty acid-hexosamine hybrids.  Glycobiology 2005;15(11):1249-1250.

2.    Lu, Q; Wang, D-S; Chen, C-S; Hu, Y-D; Chen, C-S.  Structure-based optimization of phenylbutyrate-derived histone deacetylase inhibitors.  J Med Chem 2005;48(17):5530-5535.

Radiosensitization

1.    Munshi, A; Kurland, JF; Nishikawa, T; Tanaka, T; Hobbs, ML; Tucker, SL; Ismail, S; Stevens, C; Meyn, RE.  Histone deacetylase inhibitors radiosensitize human melanoma cells by suppressing DNA repair activity.  Clin Cancer Res 2005;11(13):4912-4922.

2.    Lopez, CA; Feng, FY; Herman, JM; Nyati, MK; Lawrence, TS; Ljungman, M.  Phenylbutyrate sensitizes human glioblastoma cells lacking wild-type p53 function to ionizing radiation.  Int J Radiat Oncol Biol Phys 2007;69(1):214-220.

Resistance to Treatment

1.    Fantin, VR; Richon, VM.  Mechanisms of resistance to histone deacetylase inhibitors and their therapeutic implications.  Clin Cancer Res 2007;13(24):7237-7242.

 

Addiction

1.    Romieu, P; Host, L; Gobaille, S; Sandner, G; Aunis, D; Zwiller, J.  Histone deacetylase inhibitors decrease cocaine but not sucrose self-administration in rats.  J Neurosci 2008;28(38):9342-9348.

 

Adrenoleukodystrophy

1.    Gondcaille, C; Depreter, M; Fourcade, SU; Lecca, MR; Leclercq, S; Martin, PGP; Pineau, T; Cadepond, F; ElEtr, M; Bertrand, N; Beley, A; Duclos, S; De Craemer, D; Roels, F; Savary, SU; Bugaut, M.  Phenylbutyrate up-regulates the adrenoleukodystrophy-related gene as a nonclassical peroxisome proliferator.  J Cell Biol 2005;169(1):93-104.

 

Aging

1.    Wagner, G.  Towards a life prolonging pill? Small molecules with anti-ageing properties.  Current Drug Targets 2006;7(11):1531-1537.

2.    Burzynski SR.  Aging: gene silencing or gene activation?  Med Hypotheses 2005;64(1):201-208.

 

Alpha-Galactosidase Mutation

1.    Yam, GH-F; Roth, J; Zuber, C.  4-Phenylbutyrate rescues trafficking incompetent mutant alpha-galactosidase A without restoring its functionality.  Biochem Biophys Res Commun 2007;360(2):375-380.

 

ALS

1.    Targets in ALS: designing multidrug therapies.  Carri, MT; Grignaschi, G; Bendotti, C.  Trends Pharmacol Sci 2006;27(5):267-273.

2.    Additive neuroprotective effects of a histone deacetylase inhibitor and a catalytic antioxidant in a transgenic mouse model of amyotrophic lateral sclerosis.  Petri, S; Kiaei, M; Kipiani, K; Chen, J; Calingasan, NY; Crow, JP; Beal, MF.  Neurobiol Dis 2006;22(1):40-49.

3.    Neuroprotective agents for clinical trials in ALS: a systematic assessment.  Traynor, BJ; Bruijn, L; Conwit, R; Beal, F; O'Neill, G; Fagan, SC; Cudkowicz, ME.  Neurology 2006;67(1):20-27.

4.    Sodium phenylbutyrate prolongs survival and regulates expression of anti-apoptotic genes in transgenic amyotrophic lateral sclerosis mice.  Ryu, H; Smith, K; Camelo, SI; Carreras, I; Lee, J; Iglesias, AH; Dangond, F; Cormier, KA; Cudkowicz, ME; Brown, RH; Ferrante, RJ.  J Neurochem 2005;93(5):1087-1098.

5.    Erratum: Sodium phenylbutyrate prolongs survival and regulates expression of anti-apoptotic genes in transgenic amyotrophic lateral sclerosis mice (Journal of Neurochemistry (2005) 93 (1087-1098)).  Ryu, H; Smith, K; Camelo, SI; Carreras, I; Lee, J; Iglesias, AH; Dangond, F; Cormier, KA; Cudkowicz, ME; Brown, Jr RH; Ferrante, RJ.  J Neurochem 2006;96(3):908.

6.    Therapeutic targets for amyotrophic lateral sclerosis: Current treatments and prospects for more effective therapies.  Bruijn, LI.; Cudkowicz, M.  Expert Rev Neurother 2006;6(3):417-428.

7.    Translating preclinical insights into effective human trials in ALS.  DiBernardo, AB; Cudkowicz, ME.  Biochim Biophys Acta - Mol Basis Dis 2006;1762(11-12):1139-1149.

8.    Design, power, and interpretation of studies in the standard murine model of ALS.  Scott, S; Kranz, JE; Cole, J; Lincecum, JM; Thompson, K; Kelly, N; Bostrom, A; Theodoss, J; Al-Nakhala, BM; Vieira, FG; Ramasubbu, J; Heywood, JA.  Amyotroph Lateral Scler 2008;9(1):4-15.

9.    Safety and does escalating study of oral sodium phenylbutyrate in subjects with ALS.  Cudkowicz, ME; Andres, PL; Choudry, R; MacDonald, SA; Zhang, H; Schoenfeld, D; Ferrante, RJ.  Neurology 2007;68(12, Suppl. 1):A90.

 

Anemia

1.    A new generation of gamma globin gene inducers act on novel molecular and cellular targets.  Perrine, S; Trudel, M; Castaneda, SA.; Mankidy, R; Boosalis, MS.; Mpollo, M-SEM; White, GL; Bohacek, R; Faller, DV.  Blood 2006;108(11, Part 1):453A.

2.    Advances in the management of sickle cell disease.  Thompson, AA.  Pediatr Blood Cancer 2006;46(5):533-539.

3.    Fetal globin stimulant therapies in the beta-hemoglobinopathies: principles and current potential.  Perrine, SP.  Pediatr Ann 2008;37(5):339-346.

4.    Cucurbitacin D upregulates fetal hemoglobin expression in K562 cells and human erythroid progenitors.  Xing, H; Zhang, S; Koeffler, HP; Fung, MC.  Blood 2007;110(11, Part 2):26B-27B.

5.    Sodium phenyl butyrate downregulates endothelin-1 expression in cultured human endothelial cells: Relevance to sickle-cell disease.  Odievre, M-H; Brun, M; Krishnamoorthy, R; Lapoumeroulie, C; Elion, J.  American Journal of Hematology 2007;82(5):357-362.

6.    Pulsed-dosing with oral sodium phenylbutyrate increases hemoglobin F in a patient with sickle cell anemia.  Hines, P; Dover, GJ; Resar, LMS.  Pediatr Blood Cancer 2008;50(2):357-359.

 

Anti-Parasite Activity

1.    Scriptaid and suberoylanilide hydroxamic acid are histone deacetylase inhibitors with potent anti-Toxoplasma gondii activity in vitro.  Strobl, JS; Cassell, M; Mitchell, SM; Reilly, CM; Lindsay, DS.  J Parasitol 2007;93(3):694-700.

 

Arginase Induction

1.    Arginase induction by sodium phenylbutyrate in mouse tissues and human cell lines.  Kern, RM; Yang, Z; Kim, PS; Grody, WW; Iyer, RK; Cederbaum, SD.  Mol Genet Metab 2007;90(1):37-41.

 

Argininosuccinate Lyase Deficiency

1.    Potassium retention in patients treated for argininosuccinate lyase deficiency.  Singh, RH; Acosta, PB; Kennedy, MP; Longo, N; Elsas, LJ.  Journal of Inherited Metabolic Disease 2006;29(Suppl. 1):48.

2.    Sustained Engraftment and Tissue Enzyme Activity After Liver Cell Transplantation for Argininosuccinate Lyase Deficiency.  Stephenne, X; Najimi, M; Sibille, C; Nassogne, M; Smets, F; Sokal, EM.  Gastroenterology 2006;130(4):1317-1323.

Bladder Dysfunction

1.    A novel target for boo-induced bladder dysfunction.  Sawada, N; Yao, J; Hiramatsu, N; Hayakawa, K; Zakoji, H; Kobayashi, H; Mochizuki, T; Araki, I; Takeda, M; Kitamura, M.  European Urology Supplements 2008;7(3):265.

 

Chemical Analysis

1.    Separation and determination of optical isomers of ethyl small alpha -hydroxyphenylbutyrate by chiral HPLC.  Zhang, YY; Chen, GC.  Fenxi Ceshi Xuebao 2005;24(1):109-110.

2.    Metabolite identification using a nanoelectrospray LC-EC-array-MS integrated system.  Schiavo, S; Ebbel, E; Sharma, S; Matson, W; Kristal, BS; Hersch, S; Vouros, P.  Anal Chem 2008;80(15):5912-5923.

3.    A two-site ELISA can quantify upregulation of SMN protein by drugs for spinal muscular atrophy.  Nguyen, M; Humphrey, E; Lam, LT; Fuller, HR; Lynch, TA; Sewry, CA; Goodwin, PR; Mackenzie, AE; Morris, GE.  Neurology 2008;71(22):1757-1763.

4.    Detection of triglyceride using an iridium nano-particle catalyst based amperometric biosensor.  Liao, W-Y; Liu, C-C; Chou, T-C.  Analyst 2008;133(12):1757-1763.

5.    Multiple-layer substrate zymography for detection of several enzymes in a single sodium dodecyl sulfate gel.  Choi, N-S; Kim, B-H; Park, C-S; Han, YJ; Lee, HW; Choi, JH; Lee, S-G; Song, JJ.  Anal Biochem 2009;386(1):121-122.

 

Cholestatic Diseases

1.    Levels of plasma membrane expression in progressive and benign mutations of the bile salt export pump (Bsep/Abcb11) correlate with severity of cholestatic diseases.  Lam, P; Pearson, CL; Soroka, CJ; Xu, S; Mennone, A; Boyer, JL.  Am J Physiol Cell Physiol 2007;293(5):C1709-1716.

2.    Chemical chaperones partially reverse the mis-processing of a BRIC2 mutant of the bile salt export pump, ABCB11.  Lam, P; Soroka, CJ.; Boyer, JL.  Hepatology 2007;46(4, Suppl. S):330A.

3.    4-phenylbutyrate enhances the cell surface expression and the transport capacity of wild-type and mutated bile salt export pumps.  Hayashi, H; Sugiyama, Y.  Hepatology 2007;45(6):1506-1516.

4.    Short-chain ubiquitination is associated with the degradation rate of a cell-surface-resident bile salt export pump (BSEP/ABCB11).  Hayashi, H; Sugiyama, Y.  Mol Pharmacol 2009;75(1):143-150.

 

Citrulline Metabolism

1.    Manipulation of citrulline availability in humans.  Rougoe, C; Des Robert, C; Robins, A; Le Bacquer, O; Volteau, C; De La Cochetioere, M-F; Darmaun, D.  Am J Physiol Gastrointest Liver Physiol 2007;293(5):G1061-1067.

 

Computer Modeling

1.    Toward the computational design of diastereomeric resolving agents: an experimental and computational study of 1-phenylethylammonium-2-phenylacetate derivatives.  Karamertzanis, PG; Anandamanoharan, PR; Fernandes, P; Cains, PW; Vickers, M; Tocher, DA; Florence, AJ; Price, SL.  J Phys Chem B 2007;111(19):5326-5336.

 

Cystic Fibrosis

1.    Pharmacological induction of CFTR function in patients with cystic fibrosis: mutation-specific therapy.  Kerem, E.  Pediatr Pulmonol 2005;40(3):183-196.

2.    Novel short chain fatty acids restore chloride secretion in cystic fibrosis.  Nguyen, TD; Kim, U-S; Perrine, SP.  Biochem Biophys Res Commun 2006;342(1):245-252.

3.    Selective inhibition of endoplasmic reticulum-associated degradation rescues DeltaF508-cystic fibrosis transmembrane regulator and suppresses interleukin-8 levels: therapeutic implications.  Vij, N; Fang, S; Zeitlin, PL.  J Biol Chem 2006;281(25):17369-17378.

4.    Mutation specific therapy in CF.  Kerem, E.  Pediatr Respir Rev 2006;7(Suppl. 1):S166-S169.

5.    Pharmacoproteomics of 4-phenylbutyrate-treated IB3-1 cystic fibrosis bronchial epithelial cells.  Singh, OV; Vij, N; Mogayzel, PJ; Jozwik, C; Pollard, HB; Zeitlin, PL.  J Proteome Res  2006;5(3):562-571.

6.    Cystic fibrosis transmembrane regulator protein mutations: 'Class' opportunity for novel drug innovation.  MacDonald, KD; McKenzie, KR; Zeitlin, PL.  Pediatr Drugs 2007;9(1):1-10.

7.    Chemical rescue of deltaF508-CFTR mimics genetic repair in cystic fibrosis bronchial epithelial cells.  Singh, OV; Pollard, HB; Zeitlin, PL.  Mol Cell Proteomics 2008;7(6):1099-1110.

8.    Proinflammatory effect of sodium 4-phenylbutyrate in deltaF508-cystic fibrosis transmembrane conductance regulator lung epithelial cells: involvement of extracellular signal-regulated protein kinase 1/2 and c-Jun-NH2-terminal kinase signaling.  Roque, T; Boncoeur, E; Saint-Criq, V; Bonvin, E; Clement, A; Tabary, O; Jacquot, J.  J Pharmacol Exp Ther 2008;326(3):949-956.

Denervating Diseases

1.    New treatments for denervating diseases.  Pleasure D. J Child Neurol 2005;20(3):258-262.

Endoplasmic Reticulum Stress

1.    Mutant tamm-horsfall glycoprotein accumulation in endoplasmic reticulum induces apoptosis reversed by colchicine and sodium 4-phenylbutyrate.  Choi, SW; Ryu, OH; Choi, SJ; Song, IS; Bleyer, AJ; Hart, TC.  J Am Soc Nephrol 2005;16(10):3006-3014.

2.    Suppressive effects of 4-phenylbutyrate on the aggregation of Pael receptors and endoplasmic reticulum stress.  Kubota, K; Niinuma, Y; Kaneko, M; Okuma, Y; Sugai, M; Omura, T; Uesugi, M; Uehara, T; Hosoi, T; Nomura, Y.  J Neurochem 2006;97(5):1259-1268.

3.    Chemical chaperones reduce endoplasmic reticulum stress and prevent mutant HFE aggregate formation.  de Almeida, SF; Picarote, G; Fleming, JV; Carmo-Fonseca, M; Azevedo, JE; de Sousa, M.  J Biol Chem 2007;282(38):27905-27912.

4.    KLF5 plays an important role in endoplasmic reticulum stress response in both cardiovascular and metabolic system.  Eguchi, K; Manabe, I; Shen, H; Ohishi, Y; Fujiu, K; Takeda, N; Nagai, R.  Circulation 2007;116(16, Suppl. S):44.

5.    Involvement of endoplasmic reticulum stress in palmitate-induced endothelial cell apoptosis.  Jang, HJ; Jung, SH; Park, TJ; Jo, YE; Kim, YK; Jung, JG; Choi, S-E; Kang, Y; Kim, HJ; Kim, DJ; Lee, KW.  Diabetes 2007;56(Suppl. 1):A508-A509.

6.    Involvement of hypoxia-triggered endoplasmic reticulum stress in outlet obstruction-induced apoptosis in the urinary bladder.  Sawada, N; Yao, J; Hiramatsu, N; Hayakawa, K; Araki, I; Takeda, M; Kitamura, M.  Lab Invest 2008;88(5):553-563.

7.    BAX inhibitor-1 modulates endoplasmic reticulum stress-mediated programmed cell death in Arabidopsis.  Watanabe, N; Lam, E.  J Biol Chem 2008;283(6):3200-3210.

8.    Sodium 4-phenylbutyrate acts as a chemical chaperone on misfolded myocilin to rescue cells from endoplasmic reticulum stress and apoptosis.  Yam, GH-F; Gaplovska-Kysela, K; Zuber, C; Roth, J.  Invest Ophthalmol Vis Sci 2007;48(4):1683-1690.

9.    4-PBA(4-phenylbutyrate) reduced palmitate-induced endoplasmic reticulum stress and apoptosis in HUVEC.  Jang, H-J; Jung, J; Ha, E-S; Choi, S-E; Jung, S; Kim, H; Kim, D; Kang, Y; Lee, K-W.  Diabetologia 2007;50(Suppl. 1):S278.

10.Oxidative stress induces the endoplasmic reticulum stress and facilitates inclusion formation in cultured cells.  Hanada, S; Harada, M; Kumemura, H; Bishr Omary, M; Koga, H; Kawaguchi, T; Taniguchi, E; Yoshida, T; Hisamoto, T; Yanagimoto, C; Maeyama, M; Ueno, T; Sata, M.  J Hepatol 2007;47(1):93-102.

 

General

1.    Activity of the CHMP.  Throm, S.  Pharm Ind 2005;67(1):57-63.

2.    Activity of digestive enzymes in Scophthalmus maximus.  Fu XH; Sun M; Sun SC.  Journal of Fishery Sciences of China 2005;12(1):26-32.

3.    Novel strategy for protein exploration: high-throughput screening assisted with fuzzy neural network.  Kato, R; Nakano, H; Konishi, H; Kato, K; Koga, Y; Yamane, T; Kobayashi, T; Honda, H.  J Mol Biol 2005;351(3):683-692.

4.    Molecular cloning of enantioselective ester hydrolase from Bacillus pumilus DBRL-191.  Rasool, S; Johri, S; Riyaz-ul-Hassan, S; Maqbool, Q-u-A; Verma, V; Koul, S; Taneja, SC; Qazi, GN.  FEMS Microbiol Lett 2005;249(1):113-120.

5.    Modular assembly using sequential palladium coupling gives easy access to the SMoC class of cellular transporters.  Rebstock, A-S; Visintin, C; Leo, E; Posada, CG; Kingsbury, SR; Williams, GH; Stoeber, K; Selwood, DL.  ChemBioChem 2008;9(11):1787-1796.

6.    Waste frying oil as substrate for lipase production by Geotrichum candidum strains.  Rywinska, A; Witkowska, D; Juszczyk, P; Rymowicz, W; Kita, A.  Polish Journal of Environmental Studies 2008;17(6):925-931.

7.    The application of calcined natural dolomitic rock as a solid base catalyst in triglyceride transesterification for biodiesel synthesis.  Wilson, K; Hardacre, C; Lee, AF; Montero, JM; Shellard, L.  Green Chemistry 2008;10(6):654-659.

 

Glaucoma

1.    Russell body formation and apoptosis in myocilin-caused primary open-angle glaucoma: rescue by the chemical chaperone sodium 4-phenylbutyrate.  Roth, J; Yam, GH-F; Gaplovska-Kysela, K; Zuber, C.  FASEB Journal 2007;21(5):A182.

 

Glycosylphosphatidyl Inositol Deficiency

1.    Targeted molecular therapy for inherited glycosylphosphatidylinositol deficiency.  Almeida, AM.; Murakami, Y; Baker, A; Maeda, Y; Roberts, I; Kinoshita, T; Layton, DM; Karadimitris, A.  Blood 2006;108(11, Part 1):148A.

2.    Targeted therapy for inherited GPI deficiency.  Almeida, AM; Murakami, Y; Baker, A; Maeda, Y; Roberts, IAG; Kinoshita, T; Layton, DM; Karadimitris, A.  New Engl J Med 2007;356(16):1641-1647.

 

Huntington’s Disease

1.    Genome-wide expression profiling of human blood reveals biomarkers for Huntington's disease.  Borovecki, F; Lovrecic, L; Zhou, J; Jeong, H; Then, F; Rosas, HD; Hersch, SM; Hogarth, P; Bouzou, B; Jensen, RV; Krainc, D.  Proc Natl Acad Sci U S A 2005;102(31):11023-11028.

2.    Neuroprotective effects of phenylbutyrate in the N171-82Q transgenic mouse model of Huntington's disease.  Gardian, G; Browne, SE; Choi, D-K; Klivenyi, P; Gregorio, J; Kubilus, JK; Ryu, H; Langley, B; Ratan, RR; Ferrante, RJ; Beal, MF.  J Biol Chem 2005;280(1):556-563.

3.    A transcriptional report card from the peripheral blood: Can it measure disease progression in Huntington's disease?  Tabchy, A; Housman, D.  European Journal of Human Genetics 2006;14(6):649-650.

4.    Sodium phenylbutyrate in Huntington's disease: a dose-finding study.  Hogarth, P; Lovrecic, L; Krainc, D.  Mov Disord 2007;22(13):1962-1964.

 

Inborn Errors in Metabolism

1.    Inborn errors of metabolism: The flux from Mendelian to complex diseases.  Lanpher, B.; Brunetti-Pierri, N.; Lee, B.  Nat Rev Gen 2006;7(6):449-460.

2.    Functional and trafficking defects in ATP binding cassette A3 mutants associated with respiratory distress syndrome.  Cheong, N; Madesh, M; Gonzales, LW; Zhao, M; Yu, K; Ballard, PL; Shuman, H.  J Biol Chem 2006;281(14):9791-9800.

3.    Pharmacological treatment of congenital metabolic disorders.  Campino VA.  Aten Farm 2006;8(1):39-46.

4.    A newborn infant with hyperventilation.  Lindemann, R; Myhre, MC; Bakken, M; Fugelseth, D; Rustad, CF; Woldseth, B.  Tidsskr Nor Laegeforen 2008;128(13):1535-1536.

5.    Failure of bone morphogenetic protein receptor trafficking in pulmonary arterial hypertension: potential for rescue.  Sobolewski, A; Rudarakanchana, N; Upton, PD; Yang, J; Crilley, TK; Trembath, RC; Morrell, NW.  Hum Mol Genet 2008;17(20):3180-3190.

 

Klinefelter Syndrome

1.    An OTC deficiency 'phenocopy' in association with Klinefelter syndrome.  Swarts, L; Leisegang, F; Owen, EP; Henderson, HE.  J Inherit Metab Dis 2007;30(1):101.

 

 

 

Leucine Metabolism

1.    Acute depletion of plasma glutamine increases leucine oxidation in prednisone-treated humans.  Le Bacquer, O; Mauras, N; Welch, S; Haymond, M; Darmaun, D.  Clin Nutr 2007;26(2):231-238.

2.    Phenylbutyrate reduces plasma leucine concentrations without affecting the flux of leucine.  Marini, JC; Lanpher, B; Scaglia, F; Carter, S; Garlick, PJ; Jahoor, F; Lee, B.  FASEB Journal 2007;21(5):A335.

 

Lipoprotein Receptor

1.    Rescue of the low density lipoprotein receptor 2A mutant G544V by 4-phenylbutyrate.  Tveten, K; Holla, OL; Ranheim, T; Berge, KE; Leren, TP; Kulseth, MA.  Molecular chaperones and the heat shock response 2006;187.

2.    4-Phenylbutyrate restores the functionality of a misfolded mutant low-density lipoprotein receptor.  Tveten, K; Holla, OL; Ranheim, T; Berge, KE; Leren, TP; Kulseth, MA.  FEBS J 2007;274(8):1881-1893.

 

Lysinuric Protein Intolerance

1.    Nutrient intake in lysinuric protein intolerance.  Tanner, LM; Noantoe-Salonen, K; Venetoklis, J; Kotilainen, S; Niinikoski, H; Huoponen, K; Simell, O.  J Inherit Metab Dis 2007;30(5):716-721.

 

Mechanism of Action

1.    Does acute glutamine depletion enhance the response of glutamine synthesis to fasting in muscle in adult and old rats?  Meynial-Denis, D; Verdier, L; Mignon, M; Leclerc, J-NA; Bayle, GU; Darmaun, D.  Clin Nutr 2005;24(3):398-406.

2.    Activation of STAT-3 in CF epithelial cell by Sodium 4-Phenylbutyrate.  Suaud, L; Rubenstein, RC.  FASEB Journal 2005;19(4, Suppl. S, Part 1):A658.

3.    Cellular and molecular responses to cerebral hypoxia: Involvement of ER in neuronal death.  Nomura, Y.  Journal of Pharmacological Sciences 2005;97(Suppl. 1):6P.

4.    DNA methyltransferase (DNMT)-independent effects of DNMT inhibitors on gene expression.  Jiemjit, A; Baylin, S; Herman, J; Gore, S.  Proceedings of the American Association for Cancer Research Annual Meeting 2005;46(Suppl. S):427.

5.    Mutational analysis of 6-aminohexanoate-dimer hydrolase: Relationship between nylon oligomer hydrolytic and esterolytic activities.  Ohki, T; Wakitani, Y; Takeo, M; Yasuhira, K; Shibata, N; Higuchi, Y; Negoro, S.  FEBS Lett 2006;580(21):5054-5058.

6.    Anti-inflammatory effects of short chain fatty acids in IFN-gamma-stimulated RAW 264.7 murine macrophage cells: involvement of NF-kappaB and ERK signaling pathways.  Park, J-S; Lee, E-J; Lee, J-C; Kim, W-K; Kim, H-S.  Int Immunopharmacol 2007;7(1):70-77.

7.    Multiple molecular chaperone-mediated pharmacologic rescue of Delta F508-CFTR from ERAD.  Singh, OV; Zeitlin, PL.  FASEB Journal 2007;21(5):A420.

8.    Optimum immobilization of Candida rugosa lipase on Celite by RSM.  Chang SF; Chang SW; Yen YH; Shieh CJ.  Applied Clay Science 2007;37(1/2):67-73.

9.    Manipulating the quality control pathway in transfected cells: low temperature allows rescue of secretion-defective fibrinogen mutants.  Vu, D; Di Sanza, C; Neerman-Arbez, M.  Haematologica 2008;93(2):224-231.

10.Pro-inflammatory effect of sodium 4-phenylbutyrate in DeltaF508-CFTR lung cells: Involvement of ERK1/2 and JNK signaling.  Roque, T; Boncoeur, E; Saint-Criq, V; Bonvin, E; Clement, A; Tabary, O; Jacquot, J.  Rev Mal Respir 2008;25(9):1186.

 

Metabolism

1.    Phenylacetate catabolism in Rhodococcus sp. strain RHA1: a central pathway for degradation of aromatic compounds.  Navarro-Llorens, JM; Patrauchan, MA; Stewart, GR; Davies, JE; Eltis, LD; Mohn, WW.  J Bacteriol 2005;187(13):4497-4504.

2.    Microbial reduction of ethyl 2-oxo-4-phenylbutyrate. Searching for R-enantioselectivity. New access to the enalapril like ACE inhibitors.  de, Lacerda PSB; Ribeiro, JB; Leite, SGF; Ferrara, MA; Coelho, RB; Bon, EPS; da, Silva Lima EL; Antunes, OAC.  Tetrahedron Asymmetry 2006;17(8):1186-1188.

3.    Regio- and enantio selective reduction of methyleneketoesters mediated by Saccharomyces cerevisiae.  Clososki, GC; Milagre, CDF; Moran, PJS; Rodrigues, JAR.  Journal of Molecular Catalysis B Enzymatic 2007;48(3-4):70-76.

4.    Modulation of the activity of newly synthesized human phenylalanine hydroxylase mutant proteins by low-molecular-weight compounds.  Nascimento, C; Leandro, J; Tavares de Almeida, I; Leandro, P.  Protein J 2008;27(6):392-400.

5.    Effect of ionic liquid BMIM PF6 on asymmetric reduction of ethyl 2-oxo-4-phenylbutyrate by Saccharomyces cerevisiae.  Shi, Y-G; Fang, Y; Ren, Y-P; Wu, H-P; Guan, H-L.  J Ind Microbiol Biotechnol 2008;35(11):1419-1424.

 

N-acetylglutamate Synthase Deficiency

1.    Misleading diagnosis of partial N-acetylglutamate synthase deficiency based on enzyme measurement corrected by mutation analysis.  Heckmann, M; Wermuth, B; Heberle, J; Koch, HG; Gortner, L; Kreuder, JG.  Acta Paediatr 2005;94(1):121-124.

 

Neuroprotection

1.    Synergistic neuroprotective effects of lithium and valproic acid or other histone deacetylase inhibitors in neurons: roles of glycogen synthase kinase-3 inhibition.  Leng, Y; Liang, M-H; Ren, M; Marinova, Z; Leeds, P; Chuang, D-M.  J Neurosci 2008;28(10):2576-2588.

2.    HDAC inhibition amplifies gap junction communication in neural progenitors: potential for cell-mediated enzyme prodrug therapy.  Khan, Z; Akhtar, M; Asklund, T; Juliusson, B; Almqvist, PM; Ekstroem, TJ.  Exp Cell Res 2007;313(13):2958-2967.

3.    Endogenous alpha-synuclein is induced by valproic acid through histone deacetylase inhibition and participates in neuroprotection against glutamate-induced excitotoxicity.  Leng, Y; Chuang, D-M.  J Neurosci 2006;26(28):7502-7512.

4.    Phenylbutyrate modulates multiple neuronal survival gene expression in SMA cells.  Ganta, M; Grzeschik, SM; Heavlin, WD; Wang, CH.  Annals of Neurology 2006;60(Suppl. 10):S140.

 

 

 

 

 

Parkinson’s Disease

1.    Neurodegeneration of mouse nigrostriatal dopaminergic system induced by repeated oral administration of rotenone is prevented by 4-phenylbutyrate, a chemical chaperone.  Inden, M; Kitamura, Y; Takeuchi, H; Yanagida, T; Takata, K; Kobayashi, Y; Taniguchi, T; Yoshimoto, K; Kaneko, M; Okuma, Y; Taira, T; Ariga, H; Shimohama, S.  J Neurochem 2007;101(6):1491-1504.

2.    Neuroprotective effect of chemical chaperone 4-PBA on rotenone-induced dopaminagic neurodegeneration.  Tamaki, A; Yanagida, T; Kitamura, Y; Takata, K; Taniguchi, T.  Journal of Pharmacological Sciences 2008;106(suppl. 1):233P.

3.    Translational research of parkinsonian therapeutics.  Kitamura, Y; Taniguchi, T; Ariga, H.  J Pharm Soc Jap 2008;128:53-56.

 

Pharmacy

1.    Of orphan children and other rare medications. Part 2.  Klement A.  Chin J Dig Dis 2005;6(4):189-192.

2.    Dispensing ultra rare essential medication.   Dooms, MM; Deckx, HP; Derijdt, TF.  ASHP Midyear Clinical Meeting 2007;42.

3.    The advantage of polymer addition to a non-ionic oil in water microemulsion for the dermal delivery of progesterone.  Biruss, B; Valenta, C.  Int J Pharm 2008;349:269-273.

 

Primary Carnitine Deficiency

1.    Pharmacological rescue of carnitine transport in primary carnitine deficiency.  Amat di San Filippo, C; Pasquali, M; Longo, N.  Hum Mutat 2006;27(6):513-523.

2.    Deficiency of the carnitine transporter (OCTN2) with partial N-acetylglutamate synthase (NAGS) deficiency.  Hwu, W-L; Chien, Y-H; Tang, NLS; Law, L-K; Lin, C-Y; Lee, N-C.  J Inherit Metab Dis 2007;30(5):816.

 

Retinal Injury

1.    Retinal ischemic injury rescued by sodium 4-phenylbutyrate in a rat model.  Jeng, Y-Y; Lin, N-T; Chang, P-H; Huang, Y-P; Pang, VF; Liu, C-H; Lin, C-T.  Exp Eye Res 2007;84(3):486-492.

 

Sodium Absorption

1.    Sodium 4-phenylbutyrate upregulates ENaC and sodium absorption in T84 cells.  Iordache, C; Duszyk, M.  Exp Cell Res 2007;313(2):305-311.

2.    Modulation of epithelial sodium channel trafficking and function by sodium 4-phenylbutyrate in human nasal epithelial cells.  Prulioere-Escabasse, V; Planoes, C; Escudier, E; Fanen, P; Coste, A; Clerici, C.  J Biol Chem 2007;282(47):34048-34057.

 

Spinal Muscular Atrophy

1.    The molecular-genetic background of spinal muscular atrophy: Diagnosis, prognosis and future treatments.  Schwartz, M.  Ugeskr Laeg 2005;167(7):745-748.

2.    Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number.  Wirth, B; Brichta, L; Schrank, B; Lochmouller, H; Blick, S; Baasner, A; Heller, R.  Hum Genet 2006;119(4):422-428.

3.    Spinal muscular atrophy: from gene to therapy.  Wirth, B; Brichta, L; Hahnen, E.  Semin Pediatr Neurol 2006;13(2):121-131.

4.    Spinal muscular atrophy and therapeutic prospects.  Wirth, B; Brichta, L; Hahnen, E.  Prog Mol Subcell Biol 2006;44:109-132.

5.    Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients.  Brahe, C; Vitali, T; Tiziano, FD; Angelozzi, C; Pinto, AM; Borgo, F; Moscato, U; Bertini, E; Mercuri, E; Neri, G.  Eur J Hum Genet 2005;13(2):256-259.

6.    Clinical trials in spinal muscular atrophy.  Darras, BT; Kang, PB.  Curr Opin Pediatr 2007;19(6):675-679.

7.    Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy.  Mercuri, E; Bertini, E; Messina, S; Solari, A; D'Amico, A; Angelozzi, C; Battini, R; Berardinelli, A; Boffi, P; Bruno, C; Cini, C; Colitto, F; Kinali, M; Minetti, C; Mongini, T; Morandi, L; Neri, G; Orcesi, S; Pane, M; Pelliccioni, M; Pini, A; Tiziano, FD; Villanova, M; Vita, G; Brahe, C.  Neurology 2007;68(1):51-55.

8.    Prospective evaluation of pre-symptomatic infants with spinal muscular atrophy: Implications for early therapeutic intervention.  Swoboda, KJ.  Annals of Neurology 2007;62(Suppl. 11):S2.

9.    Survival motor neuron gene 2 silencing by DNA methylation correlates with spinal muscular atrophy disease severity and can be bypassed by histone deacetylase inhibition.  Hauke, J; Riessland, M; Lunke, S; Eyoupoglu, IY; Bloumcke, I; El-Osta, A; Wirth, B; Hahnen, E.  Hum Mol Genet 2009;18(2):304-317.

10.Drug treatment for spinal muscular atrophy types II and III.  Bosboom, WM; Vrancken, AF; van den Berg, LH; Wokke, JH; Iannaccone, St.  Cochrane Database Syst Rev 2009;1: CD006282.

11.A preliminary report on spinal muscular atrophy lymphoblastoid cell lines: are they an appropriate tool for drug screening?  Dayangaoc-Erden, D; TopaloAlu, H; Erdem-Yurter, H.  Adv Ther 2008;25(3):274-279.

 

Stability

1.    Stability of sodium phenylbutyrate in two oral liquid vehicles.  Caruthers RL.  ASHP Midyear Clinical Meeting 2006;41.

2.    Stability of extemporaneously prepared sodium phenylbutyrate oral suspensions.  Caruthers, RL; Johnson, CE.  Am J Health Syst Pharm 2007;64(14):1513-1515.

 

Synthesis

1.    Asymmetric synthesis of unnatural L-amino acids using thermophilic aromatic L-amino acid transaminase.  Cho, B-K; Seo, J-H; Kim, J; Lee, C-S; Kim, B-G.  Biotechnology and Bioprocess Engineering 2006;11(4):299-305.

2.    Preparation of enantiomerically pure (R)- and (S)-3-amino-3-phenyl-1- propanol via resolution with immobilized penicillin G acylase.  Fadnavis, NW; Radhika, KR; Vedamayee, Devi A.  Tetrahedron Asymmetry 2006;17(2):240-244.

3.    Formulation and characterization of biodegradable chitosan films for topical application of terbinafine HC1.  Ahmed, SM; Ibrahim, MA; Sarhan, HA; Amin, MA.  Bull Pharm Sci 2007;30:111-129.

4.    Creation of novel enantioselective lipases by SIMPLEX.  Koga, Y; Yamane, T; Nakano, H.  Methods Mol Biol 2007;375:165-181.

5.    Highly enantioselective sequential hydrogenation of ethyl 2-oxo-4-arylbut-3-enoate to ethyl 2-hydroxy-4-arylbutyrate.  Meng, Q; Zhu, L; Zhang, Z.  J Org Chem 2008;73(18):7209-7212.

6.    Synthesis of &gamma;-amino acid esters by 1,4-addition of deprotonated &alpha;-aminonitriles and &alpha;-(alkylideneamino)nitriles to &alpha;,&beta;-unsaturated esters.  Bergner, I; Opatz, T.  Synthesis 2007;918-928.

 

Transplantation

1.    Attenuation of primary nonfunction for syngeneic islet graft using sodium 4-phenylbutyrate.  Fu, S-H; Chen, S-T; Hsu, B R-S.  Transplant Proc 2005;37(4):1830-1831.

 

2.    The vast majority of bone-marrow-derived cells integrated into mdx muscle fibers are silent despite long-term engraftment.  Wernig, G; Janzen, V; Schefer, R; Zweyer, M; Knauf, U; Hoegemeier, O; Mundegar, RR; Garbe, S; Stier, S; Franz, T; Wernig, M; Wernig, A.  Proc Natl Acad Sci U S A 2005;102(33):11852-11857.

 

 

3.    Neurological outcome in a patient with a null ornithine transcarbamylase genotype following liver transplantation at 6 weeks of age.  Orfanelli, L; Greene, C; Glass, P; Gaillard, WD; Vezina, LG; Issacs, J; Higgs, J; Tuchman, M; Dunn, S; Berry, GT.  Journal of Inherited Metabolic Disease 2006;29(Suppl. 1):31.

 

4.    Enhancing engraftment of islets using perioperative sodium 4-phenylbutyrate.  Hsu, BR-S; Chen, S-T; Fu, S-H.  Int Immunopharmacol 2006;6(13-14):1952-1959.

 

Urea Cycle Disorders

1.    Orphan drug: Carglumic acid for the treatment of urea cycle disorders.  Dtsch Apoth Ztg 2005;145(1):44-46.

2.    Sodium 4-phenylbutyrate protects against liver ischemia reperfusion injury by inhibition of endoplasmic reticulum-stress mediated apoptosis.  Vilatoba, M; Eckstein, C; Bilbao, G; Smyth, CA; Jenkins, S; Thompson, JA; Eckhoff, DE; Contreras, JL.  Surgery 2005;138(2):342-351.

3.    Long-term outcome of urea cycle disorders.  Bachmann, C.  Acta Gastroenterol Belg 2005;68(4):466-468.

4.    The pattern of intravenous drug administration during the transfer of critically ill children by a specialist transport team.  Saha, AS; Langridge, P; Playfor, SD.  Paediatr Anaesth 2006;16(10):1063-1067.

5.    Successful extracorporeal treatment of a male with hyperammonaemic coma.  Haller, M; Boulanger, AH-L; Sass, JO; Brandis, M; Zimmerhackl, LB.  Nephrology Dialysis Transplantation 2005;20(2):453-455.

6.    Outcomes among male OTC patients treated with ammonaps (R).  Wuebbels, BH.  Journal of Inherited Metabolic Disease 2006;29(Suppl. 1):129.

7.    Urea cycle disorders in adult patients.  Maillot, F; Crenn, P.  Revue Neurologique (Paris) 2007;163(10):897-903.

8.    A review of biochemical outcomes of adults with OTC deficiency.  Bhattacharya, K; Briddon, A; Lee, PJ.  Journal of Inherited Metabolic Disease 2007;30(Suppl. 1):82.

9.    Cross-sectional multicenter study of patients with urea cycle disorders in the United States.  Tuchman, M; Lee, B; Lichter-Konecki, U; Summar, ML; Yudkoff, M; Cederbaum, SD; Kerr, DS; Diaz, GA; Seashore, MR; Lee, H-S; McCarter, RJ; Krischer, JP; Batshaw, ML; Urea Cycle Disorders Consortium of the Rare Diseases Clinical Research Network.  Mol Genet Metab 2008;94(4):397-402.

10.Hypothesis: proposals for the management of a neonate at risk of hyperammonaemia due to a urea cycle disorder.  Leonard, JV; Ward, P; Martin, P; Morris, AAM.  Eur J Pediatr 2008;167(3):305-309.

11.L-ornithine phenylacetate reduces arterial ammonia, improves brain osmolytes and reduces brain water in a bile duct ligated pat model of cirrhosis.  Wright, GA; Davies, NA; Ytrebo, LM; Stadlbauer, V; Fuskevag, O-M; Zwingmann, C; Hodges, S; Jalan, R.  Hepatology 2007;46(4, Suppl. S):604A-605A.

12.Preliminary data on adult patients with urea cycle disorders (UCD) in an open-label, switch-over, dose-escalation study comparing a new ammonia scavenger, glyceryl tri(4-phenylbutyrate) (HPN-100), to buphenyl (sodium phenylbutyrate (PBA).  Lee, B; Garovoy, MR; Gargosky, SE; Berry, SA.  Journal of Inherited Metabolic Disease 2008;31(Suppl. 1):91.

13.Sodium phenylbutyrate (Ammonaps) in the treatment of patients with urea cycle disorders - European post-approval data.  Dobbelaere, DD.  Journal of Inherited Metabolic Disease 2008;31(Suppl. 1):92.