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 

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.


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.


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.


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.


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.


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.



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.



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.



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.



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.



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.



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.



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.



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.



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.



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.



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.



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.



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.