J.L. Kakkis: search on Z-Library

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51

The Randomized Blind Start Trial: Evaluation of A New Study Design for Assessing Clinical Outcomes in Rare and Heterogeneous Patient Populations

Signorovitch, J., Ayyagari, R., Kakkis, E.

Journal:

Value in Health

Year:

2014

Language:

english

File:

PDF, 61 KB

english, 2014

52

Serum Neural Cell Adhesion Molecule Is Hyposialylated in Hereditary Inclusion Body Myopathy

Valles-Ayoub, Yadira, Esfandiarifard, Saghi, Sinai, Pedram, Carbajo, Rosangela, Khokher, Zeshan, No, Daniel, Pietruszka, Marvin, Darvish, Babak, Kakkis, Emil, Darvish, Daniel

Journal:

Genetic Testing and Molecular Biomarkers

Year:

2012

Language:

english

File:

PDF, 194 KB

english, 2012

53

Enzyme replacement therapy for the mucopolysaccharide storage disorders

Journal:

Expert Opinion on Investigational Drugs

Year:

2002

Language:

english

File:

PDF, 192 KB

english, 2002

54

Carbohydrate Structures of Recombinant Human -L-Iduronidase Secreted by Chinese Hamster Ovary Cells

Zhao, K.-W., Faull, K. F., Kakkis, E. D., Neufeld, E. F.

Journal:

Journal of Biological Chemistry

Year:

1997

Language:

english

File:

PDF, 837 KB

english, 1997

55

Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I

Kakkis, E., Lester, T., Yang, R., Tanaka, C., Anand, V., Lemontt, J., Peinovich, M., Passage, M.

Journal:

Proceedings of the National Academy of Sciences

Year:

2004

Language:

english

File:

PDF, 808 KB

english, 2004

56

From The Cover: Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII

Vogler, C., Levy, B., Grubb, J. H., Galvin, N., Tan, Y., Kakkis, E., Pavloff, N., Sly, W. S.

Journal:

Proceedings of the National Academy of Sciences

Year:

2005

Language:

english

File:

PDF, 613 KB

english, 2005

57

Near-complete pathological response with preoperative chemotherapy in a patient with metaplastic breast carcinoma

Amini, Arya, Kakkis, Jane, Reitherman, Richard, Ibarra, Julio, Sanati, Homayoon

Journal:

Anti-Cancer Drugs

Year:

2013

Language:

english

File:

PDF, 247 KB

english, 2013

58

First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient

Fox, Joyce E., Volpe, Linda, Bullaro, Josephine, Kakkis, Emil D., Sly, William S.

Journal:

Molecular Genetics and Metabolism

Year:

2015

Language:

english

File:

PDF, 394 KB

english, 2015

59

Strong transcriptional activation of translocated c-myc genes occurs without a strong nearby enhancer or promoter

Kakkis, Emil, Mercola, Mark, Calame, Kathryn

Journal:

Nucleic Acids Research

Year:

1988

Language:

english

File:

PDF, 2.41 MB

english, 1988

60

Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment—A retrospective chart review

Vockley, Jerry, Marsden, Deborah, McCracken, Elizabeth, DeWard, Stephanie, Barone, Amanda, Hsu, Kristen, Kakkis, Emil

Journal:

Molecular Genetics and Metabolism

Year:

2015

Language:

english

File:

PDF, 1.29 MB

english, 2015

61

Enzyme replacement therapy (ERT) for mucopolysaccharidosis VII (MPS VII; Sly syndrome) reduces lysosomal storage in a 36-week phase 1/2 clinical study

Jones, Simon A., Ghosh, Arunabha, Breen, Catherine, Kakkis, Emil D., Sly, William S.

Journal:

Molecular Genetics and Metabolism

Year:

2015

Language:

english

File:

PDF, 55 KB

english, 2015

62

Enzyme replacement therapy with investigational rhGUS in an infant with non-immune hydrops fetalis and mucopolysaccharidosis type VII

Lau, Heather A., Parmar, Swapnil, Kazachkov, Mikhail, Shah, Rasikial, Wells, John, Yachelevich, Naomi, Chopra, Arun, Haller, Christine, Kakkis, Emil

Journal:

Molecular Genetics and Metabolism

Year:

2016

Language:

english

File:

PDF, 49 KB

english, 2016

63

Evidence of early bone response after initiation of enzyme replacement therapy in a 3year old patient with MPS VII

Lau, Heather A., Parmar, Swapnil, Ferraris, Marissa, Haller, Christine, Agarwal, Sunil, Kakkis, Emil

Journal:

Molecular Genetics and Metabolism

Year:

2016

Language:

english

File:

PDF, 48 KB

english, 2016

64

Enzyme Replacement in a Canine Model of Hurler Syndrome

Robert M. Shull, Emil D. Kakkis, Michael F. McEntee, Stephen A. Kania, Adam J. Jonas and Elizabeth F. Neufeld

Journal:

Proceedings of the National Academy of Sciences

Year:

1994

Language:

english

File:

PDF, 1.48 MB

english, 1994

65

Chronic Administration of the Nitric Oxide Synthase Inhibitor, L-NAME, Increases Circulating Endothelin Levels in Guinea Pigs

Miller, Mark J. S., Munshi, Upender K., Zhang, Xiao-Jing, Sadowska-Krowicka, Halina, Savage, Paula, Kakkis, Jane L., Eloby-Childress, Sandra, Clark, David A., Bustamante, Sergio A., Jeng, Arco Y.

Journal:

Year:

1995

Language:

english

File:

PDF, 623 KB

english, 1995

66

Corrigendum to ‘Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment—A retrospective chart review’, Molecular Genetics and Metabolism (2015) 53–60

Vockley, Jerry, Marsden, Deborah, McCracken, Elizabeth, DeWard, Stephanie, Barone, Amanda, Hsu, Kristen, Kakkis, Emil

Journal:

Molecular Genetics and Metabolism

Year:

2015

Language:

english

File:

PDF, 111 KB

english, 2015

67

Hemodynamic Changes after Protamine Administration

Welsby, Ian J., Newman, Mark F., Phillips-Bute, Barbara, Messier, Robert H., Kakkis, Emil D., Stafford-Smith, Mark

Journal:

Year:

2005

Language:

english

File:

PDF, 574 KB

english, 2005

68

A novel approach to characterization and categorization of infusion reactions associated with ERT using adverse physiology related groups

Haller, Christine, Agarwal, Sunil, Kakkis, Emil

Journal:

Molecular Genetics and Metabolism

Year:

2016

Language:

english

File:

PDF, 53 KB

english, 2016

69

Enzyme replacement in a canine model of Hurler syndrome.

Shull, R. M., Kakkis, E. D., McEntee, M. F., Kania, S. A., Jonas, A. J., Neufeld, E. F.

Journal:

Proceedings of the National Academy of Sciences

Year:

1994

Language:

english

File:

PDF, 2.36 MB

english, 1994

70

An active chromatin structure acquired by translocated c-myc genes.

Kakkis, E, Prehn, J, Calame, K

Journal:

Molecular and Cellular Biology

Year:

1986

Language:

english

File:

PDF, 1.31 MB

english, 1986

71

A plasmacytoma-specific factor binds the c-myc promoter region.

Kakkis, E., Calame, K.

Journal:

Proceedings of the National Academy of Sciences

Year:

1987

Language:

english

File:

PDF, 1.49 MB

english, 1987

72

Patients as key partners in rare disease drug development

Bronstein, Max G., Kakkis, Emil D.

Journal:

Nature Reviews Drug Discovery

Year:

2016

Language:

english

File:

PDF, 111 KB

english, 2016

73

Dopaminergic modulation of the renal effect of arginine-vasopressin in water-loaded rats.

KOYAMA, Shigeru, SASAKI, Masato, SETOYAMA, Toshiyuki, TAKAHASHI, Kazuhito, TOGASHI, Kazutomo, SUZUKI, Kohki, KAKKI, Nobuyuki, IMAI, Masashi

Journal:

The Japanese Journal of Pharmacology

Year:

1985

Language:

english

File:

PDF, 318 KB

english, 1985

74

A novel, randomized, placebo-controlled, blind-start, single-crossover phase 3 study to assess the efficacy and safety of UX003 (rhGUS) enzyme replacement therapy in patients with MPS VII

Harmatz, Paul, Whitley, Chester B., Wang, Raymond Y., Bauer, Mislen, Song, Wenjie, Jacobs, Karen, Schwartz, Erin, Haller, Christine, Kakkis, Emil

Journal:

Molecular Genetics and Metabolism

Year:

2017

Language:

english

File:

PDF, 51 KB

english, 2017

75

A phase 3 randomized, double blind, placebo-controlled study to evaluate the efficacy and safety of sialic acid extended-release tablets in patients with GNE myopathy (GNEM)

Lochmüller, H., Behin, A., Caraco, Y., Lau, H., Mirabella, M., Tournev, I., Tarnopolsky, M., Pogoryelova, O., Shah, J., Koutsoubos, T., Skrinar, A., Mansbach, H., Kakkis, E., Mozaffar, T.

Journal:

Neuromuscular Disorders

Year:

2017

Language:

english

File:

PDF, 242 KB

english, 2017

76

Phenotypic stratification and genotype–phenotype correlation in a heterogeneous, international cohort of GNE myopathy patients: First report from the GNE myopathy Disease Monitoring Program, registry portion

Pogoryelova, Oksana, Cammish, Phillip, Mansbach, Hank, Argov, Zohar, Nishino, Ichizo, Skrinar, Alison, Chan, Yiumo, Nafissi, Shahriar, Shamshiri, Hosein, Kakkis, Emil, Lochmüller, Hanns

Journal:

Neuromuscular Disorders

Year:

2017

Language:

english

File:

PDF, 1.48 MB

english, 2017

77

Aceneuramic Acid Extended Release Administration Maintains Upper Limb Muscle Strength in a 48-week Study of Subjects with GNE Myopathy: Results from a Phase 2, Randomized, Controlled Study

Argov, Zohar, Caraco, Yoseph, Lau, Heather, Pestronk, Alan, Shieh, Perry B., Skrinar, Alison, Koutsoukos, Tony, Ahmed, Ruhi, Martinisi, Julia, Kakkis, Emil

Journal:

Journal of Neuromuscular Diseases

Year:

2016

Language:

english

File:

PDF, 680 KB

english, 2016

78

Vestronidase alfa: Recombinant human Î²-glucuronidase as an enzyme replacement therapy for MPS VII

Cadaoas, Jaclyn, Boyle, Gabrielle, Jungles, Steven, Cullen, Sean, Vellard, Michel, Grubb, Jeffrey H., Jurecka, Agnieszka, Sly, William, Kakkis, Emil

Journal:

Molecular Genetics and Metabolism

Year:

2020

File:

PDF, 1.89 MB

2020

79

Conventional and Unconventional Therapeutic Strategies for Sialidosis Type I

Mosca, Rosario, Vlekkert, Diantha van de, Campos, Yvan, Fremuth, Leigh E., Cadaoas, Jaclyn, Koppaka, Vish, Kakkis, Emil, Tifft, Cynthia, Toro, Camilo, Allievi, Simona, Gellera, Cinzia, Canafoglia, Lau

Journal:

Journal of Clinical Medicine

Year:

2020

File:

PDF, 1.63 MB

2020

80

Urinary glycosaminoglycans as a potential biomarker for evaluating treatment efficacy in subjects with mucopolysaccharidoses

Kakkis, Emil, Marsden, Deborah

Journal:

Molecular Genetics and Metabolism

Year:

2020

File:

PDF, 1.08 MB

2020

81

Treatments Administered to the First 9152 Reported Cases of COVID-19: A Systematic Review

Fajgenbaum, David C., Khor, Johnson S., Gorzewski, Alexander, Tamakloe, Mark-Avery, Powers, Victoria, Kakkis, Joseph J., Repasky, Mileva, Taylor, Anne, Beschloss, Alexander, Hernandez-Miyares, Laura,

Journal:

Infectious Diseases and Therapy

Year:

2020

File:

PDF, 897 KB

2020

82

Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses

Jones, Simon A., Marsden, Deborah, Koutsoukos, Tony, Sniadecki, Jennifer, Tylee, Karen, Phillippo, Sarah, Kakkis, Emil

Journal:

Molecular Genetics and Metabolism

Year:

2020

File:

PDF, 410 KB

2020

83

MetalâTemplated Design of Chemically Switchable Protein Assemblies with HighâAffinity Coordination Sites

Tezcan, F. Akif, Kakkis, Albert, Gagnon, Derek, Esselborn, Julian, Britt, R. David

Journal:

Angewandte Chemie International Edition

Year:

2020

File:

PDF, 2.71 MB

2020

84

MetalâTemplated Design of Chemically Switchable Protein Assemblies with HighâAffinity Coordination Sites

Tezcan, F. Akif, Kakkis, Albert, Gagnon, Derek, Esselborn, Julian, Britt, R. David

Journal:

Angewandte Chemie

Year:

2020

File:

PDF, 2.71 MB

2020