Emil Kakkis

Title(s)Assistant Adjunct Professor, Pediatrics
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    1. Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques. Gene Ther. 2024 Mar; 31(3-4):128-143. Kistner A, Chichester JA, Wang L, Calcedo R, Greig JA, Cardwell LN, Wright MC, Couthouis J, Sethi S, McIntosh BE, McKeever K, Wadsworth S, Wilson JM, Kakkis E, Sullivan BA. PMID: 37833563; PMCID: PMC10940161.
      View in: PubMed   Mentions:    Fields:    Translation:HumansAnimalsCells
    2. The transformation of drug development for the 21st century: Time for a change. Mol Genet Metab. 2022 Sep-Oct; 137(1-2):107-113. Kakkis ED. PMID: 35985171.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    3. Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression. Mol Ther Methods Clin Dev. 2022 Mar 10; 24:292-305. Wang L, Warzecha CC, Kistner A, Chichester JA, Bell P, Buza EL, He Z, Pampena MB, Couthouis J, Sethi S, McKeever K, Betts MR, Kakkis E, Wilson JM, Wadsworth S, Sullivan BA. PMID: 35211641; PMCID: PMC8841522.
      View in: PubMed   Mentions: 9  
    4. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 04 19; 16(1):183. Tandon PK, Kakkis ED. PMID: 33874971; PMCID: PMC8054393.
      View in: PubMed   Mentions: 8     Fields:    Translation:Humans
    5. Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders. Orphanet J Rare Dis. 2021 03 20; 16(1):141. Lochmüller H, Ramirez AN, Kakkis E. PMID: 33743771; PMCID: PMC7980582.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    6. Results from a 3-year Non-interventional, Observational Disease Monitoring Program in Adults with GNE Myopathy. J Neuromuscul Dis. 2021; 8(2):225-234. Lochmüller H, Behin A, Tournev I, Tarnopolsky M, Horváth R, Pogoryelova O, Shah J, Koutsoukos T, Skrinar A, Kakkis E, Bedrosian CL, Mozaffar T. PMID: 33459658; PMCID: PMC8075380.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansCTClinical Trials
    7. Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept. Mol Ther Methods Clin Dev. 2021 Mar 12; 20:191-203. Cadaoas J, Hu H, Boyle G, Gomero E, Mosca R, Jayashankar K, Machado M, Cullen S, Guzman B, van de Vlekkert D, Annunziata I, Vellard M, Kakkis E, Koppaka V, d'Azzo A. PMID: 33426146; PMCID: PMC7782203.
      View in: PubMed   Mentions: 7  
    8. Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses. Mol Genet Metab. 2020 08; 130(4):255-261. Jones SA, Marsden D, Koutsoukos T, Sniadecki J, Tylee K, Phillippo S, Kakkis E. PMID: 32563631.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    9. Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII. Mol Genet Metab. 2020 05; 130(1):65-76. Cadaoas J, Boyle G, Jungles S, Cullen S, Vellard M, Grubb JH, Jurecka A, Sly W, Kakkis E. PMID: 32192868.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
    10. Conventional and Unconventional Therapeutic Strategies for Sialidosis Type I. J Clin Med. 2020 03 04; 9(3). Mosca R, van de Vlekkert D, Campos Y, Fremuth LE, Cadaoas J, Koppaka V, Kakkis E, Tifft C, Toro C, Allievi S, Gellera C, Canafoglia L, Visser G, Annunziata I, d'Azzo A. PMID: 32143456; PMCID: PMC7141319.
      View in: PubMed   Mentions: 10  
    11. Urinary glycosaminoglycans as a potential biomarker for evaluating treatment efficacy in subjects with mucopolysaccharidoses. Mol Genet Metab. 2020 05; 130(1):7-15. Kakkis E, Marsden D. PMID: 32224020.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimals
    12. A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy. Neurology. 2019 04 30; 92(18):e2109-e2117. Lochmüller H, Behin A, Caraco Y, Lau H, Mirabella M, Tournev I, Tarnopolsky M, Pogoryelova O, Woods C, Lai A, Shah J, Koutsoukos T, Skrinar A, Mansbach H, Kakkis E, Mozaffar T. PMID: 31036580; PMCID: PMC6512882.
      View in: PubMed   Mentions: 24     Fields:    Translation:HumansCTClinical Trials
    13. Results from a 78-week, single-arm, open-label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long-chain fatty acid oxidation disorders (LC-FAOD). J Inherit Metab Dis. 2019 01; 42(1):169-177. Vockley J, Burton B, Berry GT, Longo N, Phillips J, Sanchez-Valle A, Tanpaiboon P, Grunewald S, Murphy E, Bowden A, Chen W, Chen CY, Cataldo J, Marsden D, Kakkis E. PMID: 30740733; PMCID: PMC6348052.
      View in: PubMed   Mentions: 25     Fields:    Translation:HumansCellsCTClinical Trials
    14. Burosumab Therapy in Children with X-Linked Hypophosphatemia. N Engl J Med. 2018 05 24; 378(21):1987-1998. Carpenter TO, Whyte MP, Imel EA, Boot AM, Högler W, Linglart A, Padidela R, Van't Hoff W, Mao M, Chen CY, Skrinar A, Kakkis E, San Martin J, Portale AA. PMID: 29791829.
      View in: PubMed   Mentions: 162     Fields:    Translation:HumansCTClinical Trials
    15. A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease. Mol Genet Metab. 2018 04; 123(4):488-494. Harmatz P, Whitley CB, Wang RY, Bauer M, Song W, Haller C, Kakkis E. PMID: 29478819.
      View in: PubMed   Mentions: 25     Fields:    Translation:HumansCTClinical Trials
    16. 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. Neuromuscul Disord. 2018 02; 28(2):158-168. Pogoryelova O, Cammish P, Mansbach H, Argov Z, Nishino I, Skrinar A, Chan Y, Nafissi S, Shamshiri H, Kakkis E, Lochmüller H. PMID: 29305133; PMCID: PMC5857291.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    17. Substantial deficiency of free sialic acid in muscles of patients with GNE myopathy and in a mouse model. PLoS One. 2017; 12(3):e0173261. Chan YM, Lee P, Jungles S, Morris G, Cadaoas J, Skrinar A, Vellard M, Kakkis E. PMID: 28267778; PMCID: PMC5340369.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansAnimals
    18. UX007 for the treatment of long chain-fatty acid oxidation disorders: Safety and efficacy in children and adults following 24weeks of treatment. Mol Genet Metab. 2017 04; 120(4):370-377. Vockley J, Burton B, Berry GT, Longo N, Phillips J, Sanchez-Valle A, Tanpaiboon P, Grunewald S, Murphy E, Humphrey R, Mayhew J, Bowden A, Zhang L, Cataldo J, Marsden DL, Kakkis E. PMID: 28189603.
      View in: PubMed   Mentions: 29     Fields:    Translation:HumansCTClinical Trials
    19. Patients as key partners in rare disease drug development. Nat Rev Drug Discov. 2016 11; 15(11):731-732. Bronstein MG, Kakkis ED. PMID: 27444223.
      View in: PubMed   Mentions: 10     Fields:    Translation:Humans
    20. Accessing the accelerated approval pathway for rare disease therapeutics. Nat Biotechnol. 2016 Apr; 34(4):380-3. Kakkis ED, Kowalcyk S, Bronstein MG. PMID: 27054988.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    21. 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. J Neuromuscul Dis. 2016 03 03; 3(1):49-66. Argov Z, Caraco Y, Lau H, Pestronk A, Shieh PB, Skrinar A, Koutsoukos T, Ahmed R, Martinisi J, Kakkis E. PMID: 27854209; PMCID: PMC5271423.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansCTClinical Trials
    22. The 21st Century Cures Act. N Engl J Med. 2015 10 22; 373(17):1678. Kakkis E, Bronstein MG. PMID: 26488712.
      View in: PubMed   Mentions:    Fields:    Translation:HumansPHPublic Health
    23. 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. Mol Genet Metab. 2015 Sep-Oct; 116(1-2):53-60. Vockley J, Marsden D, McCracken E, DeWard S, Barone A, Hsu K, Kakkis E. PMID: 26116311; PMCID: PMC4561603.
      View in: PubMed   Mentions: 45     Fields:    Translation:HumansCells
    24. Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints. Orphanet J Rare Dis. 2015 Feb 10; 10:16. Kakkis ED, O'Donovan M, Cox G, Hayes M, Goodsaid F, Tandon PK, Furlong P, Boynton S, Bozic M, Orfali M, Thornton M. PMID: 25757705; PMCID: PMC4347559.
      View in: PubMed   Mentions: 21     Fields:    Translation:Humans
    25. First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient. Mol Genet Metab. 2015 Feb; 114(2):203-8. Fox JE, Volpe L, Bullaro J, Kakkis ED, Sly WS. PMID: 25468648; PMCID: PMC4360956.
      View in: PubMed   Mentions: 35     Fields:    Translation:Humans
    26. The Randomized Blind Start Trial: Evaluation of A New Study Design for Assessing Clinical Outcomes in Rare and Heterogeneous Patient Populations. Value Health. 2014 Nov; 17(7):A580. Signorovitch J, Ayyagari R, Kakkis E. PMID: 27201958.
      View in: PubMed   Mentions: 1     Fields:    
    27. Mannose 6-phosphate conjugation is not sufficient to allow induction of immune tolerance to phenylalanine ammonia-lyase in dogs. JIMD Rep. 2013; 8:63-72. Vera M, Lester T, Zhao B, Tiger P, Clarke S, Tippin BL, Passage MB, Le SQ, Femenia J, Lemontt JF, Kakkis ED, Dickson PI. PMID: 23430522; PMCID: PMC3565636.
      View in: PubMed   Mentions:
    28. Serum neural cell adhesion molecule is hyposialylated in hereditary inclusion body myopathy. Genet Test Mol Biomarkers. 2012 May; 16(5):313-7. Valles-Ayoub Y, Esfandiarifard S, Sinai P, Carbajo R, Khokher Z, No D, Pietruszka M, Darvish B, Kakkis E, Darvish D. PMID: 22085395.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansAnimals
    29. The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases. Orphanet J Rare Dis. 2011 Jul 06; 6:49. Miyamoto BE, Kakkis ED. PMID: 21733145; PMCID: PMC3149566.
      View in: PubMed   Mentions: 21     Fields:    Translation:HumansAnimals
    30. Arterial pathology in canine mucopolysaccharidosis-I and response to therapy. Lab Invest. 2011 May; 91(5):665-74. Lyons JA, Dickson PI, Wall JS, Passage MB, Ellinwood NM, Kakkis ED, McEntee MF. PMID: 21383673; PMCID: PMC3084338.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansAnimals
    31. Research challenges in central nervous system manifestations of inborn errors of metabolism. Mol Genet Metab. 2011 Mar; 102(3):326-38. Dickson PI, Pariser AR, Groft SC, Ishihara RW, McNeil DE, Tagle D, Griebel DJ, Kaler SG, Mink JW, Shapiro EG, Bjoraker KJ, Krivitzky L, Provenzale JM, Gropman A, Orchard P, Raymond G, Cohen BH, Steiner RD, Goldkind SF, Nelson RM, Kakkis E, Patterson MC. PMID: 21176882; PMCID: PMC3040279.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansAnimals
    32. Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice. PLoS One. 2010 Aug 16; 5(8):e12194. Dvorak-Ewell M, Wendt D, Hague C, Christianson T, Koppaka V, Crippen D, Kakkis E, Vellard M. PMID: 20808938; PMCID: PMC2922370.
      View in: PubMed   Mentions: 22     Fields:    Translation:HumansAnimalsCells
    33. Intrathecal administration of recombinant human N-acetylgalactosamine 4-sulfatase to a MPS VI patient with pachymeningitis cervicalis. Mol Genet Metab. 2010 Apr; 99(4):346-50. Muñoz-Rojas MV, Horovitz DD, Jardim LB, Raymundo M, Llerena JC, de Magalhães Tde S, Vieira TA, Costa R, Kakkis E, Giugliani R. PMID: 20036175.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    34. Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen. Mol Genet Metab. 2010 Feb; 99(2):132-41. Auclair D, Finnie J, White J, Nielsen T, Fuller M, Kakkis E, Cheng A, O'Neill CA, Hopwood JJ. PMID: 19896877.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansAnimals
    35. Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs. J Inherit Metab Dis. 2009 Dec; 32 Suppl 1:S253-8. Passage MB, Krieger AW, Peinovich MC, Lester T, Le SQ, Dickson PI, Kakkis ED. PMID: 19562502; PMCID: PMC2889197.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansAnimals
    36. Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study. J Pediatr. 2009 May; 154(5):700-7. Trefz FK, Burton BK, Longo N, Casanova MM, Gruskin DJ, Dorenbaum A, Kakkis ED, Crombez EA, Grange DK, Harmatz P, Lipson MH, Milanowski A, Randolph LM, Vockley J, Whitley CB, Wolff JA, Bebchuk J, Christ-Schmidt H, Hennermann JB, Sapropterin Study Group. PMID: 19261295.
      View in: PubMed   Mentions: 64     Fields:    Translation:HumansCTClinical Trials
    37. Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics. 2009 Jan; 123(1):229-40. Clarke LA, Wraith JE, Beck M, Kolodny EH, Pastores GM, Muenzer J, Rapoport DM, Berger KI, Sidman M, Kakkis ED, Cox GF. PMID: 19117887.
      View in: PubMed   Mentions: 129     Fields:    Translation:Humans
    38. A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I. Mol Genet Metab. 2009 Jan; 96(1):13-9. Giugliani R, Rojas VM, Martins AM, Valadares ER, Clarke JT, Góes JE, Kakkis ED, Worden MA, Sidman M, Cox GF. PMID: 19038563.
      View in: PubMed   Mentions: 20     Fields:    Translation:Humans
    39. Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression. Am J Med Genet A. 2008 Oct 01; 146A(19):2538-44. Munoz-Rojas MV, Vieira T, Costa R, Fagondes S, John A, Jardim LB, Vedolin LM, Raymundo M, Dickson PI, Kakkis E, Giugliani R. PMID: 18792977.
      View in: PubMed   Mentions: 37     Fields:    Translation:Humans
    40. Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I. J Clin Invest. 2008 Aug; 118(8):2868-76. Dickson P, Peinovich M, McEntee M, Lester T, Le S, Krieger A, Manuel H, Jabagat C, Passage M, Kakkis ED. PMID: 18654665; PMCID: PMC2483682.
      View in: PubMed   Mentions: 59     Fields:    Translation:HumansAnimalsCells
    41. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase). Pediatrics. 2007 Jul; 120(1):e37-46. Wraith JE, Beck M, Lane R, van der Ploeg A, Shapiro E, Xue Y, Kakkis ED, Guffon N. PMID: 17606547.
      View in: PubMed   Mentions: 105     Fields:    Translation:HumansCTClinical Trials
    42. Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid. Mol Genet Metab. 2007 May; 91(1):61-8. Dickson P, McEntee M, Vogler C, Le S, Levy B, Peinovich M, Hanson S, Passage M, Kakkis E. PMID: 17321776; PMCID: PMC3009387.
      View in: PubMed   Mentions: 72     Fields:    Translation:HumansAnimals
    43. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. Mol Genet Metab. 2007 Feb; 90(2):171-80. Sifuentes M, Doroshow R, Hoft R, Mason G, Walot I, Diament M, Okazaki S, Huff K, Cox GF, Swiedler SJ, Kakkis ED. PMID: 17011223.
      View in: PubMed   Mentions: 98     Fields:    Translation:HumansCells
    44. Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice. Gene Ther. 2006 Jun; 13(11):917-25. Watson G, Bastacky J, Belichenko P, Buddhikot M, Jungles S, Vellard M, Mobley WC, Kakkis E. PMID: 16482204.
      View in: PubMed   Mentions: 48     Fields:    Translation:AnimalsCells
    45. Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr. 2006 Apr; 148(4):533-539. Harmatz P, Giugliani R, Schwartz I, Guffon N, Teles EL, Miranda MC, Wraith JE, Beck M, Arash L, Scarpa M, Yu ZF, Wittes J, Berger KI, Newman MS, Lowe AM, Kakkis E, Swiedler SJ, MPS VI Phase 3 Study Group. PMID: 16647419.
      View in: PubMed   Mentions: 125     Fields:    Translation:HumansCTClinical Trials
    46. Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII. Proc Natl Acad Sci U S A. 2005 Oct 11; 102(41):14777-82. Vogler C, Levy B, Grubb JH, Galvin N, Tan Y, Kakkis E, Pavloff N, Sly WS. PMID: 16162667; PMCID: PMC1253584.
      View in: PubMed   Mentions: 81     Fields:    Translation:HumansAnimalsCells
    47. Penetration, diffusion, and uptake of recombinant human alpha-L-iduronidase after intraventricular injection into the rat brain. Mol Genet Metab. 2005 Sep-Oct; 86(1-2):141-9. Belichenko PV, Dickson PI, Passage M, Jungles S, Mobley WC, Kakkis ED. PMID: 16006167.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimalsCells
    48. Hemodynamic changes after protamine administration: association with mortality after coronary artery bypass surgery. Anesthesiology. 2005 Feb; 102(2):308-14. Welsby IJ, Newman MF, Phillips-Bute B, Messier RH, Kakkis ED, Stafford-Smith M. PMID: 15681944.
      View in: PubMed   Mentions: 15     Fields:    Translation:Humans
    49. Identification and molecular characterization of alpha-L-iduronidase mutations present in mucopolysaccharidosis type I patients undergoing enzyme replacement therapy. Hum Mutat. 2004 Sep; 24(3):199-207. Yogalingam G, Guo XH, Muller VJ, Brooks DA, Clements PR, Kakkis ED, Hopwood JJ. PMID: 15300847.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansAnimalsCells
    50. Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I. Mol Genet Metab. 2004 Sep-Oct; 83(1-2):163-74. Kakkis E, McEntee M, Vogler C, Le S, Levy B, Belichenko P, Mobley W, Dickson P, Hanson S, Passage M. PMID: 15464431.
      View in: PubMed   Mentions: 60     Fields:    Translation:HumansAnimalsCells
    51. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr. 2004 May; 144(5):581-8. Wraith JE, Clarke LA, Beck M, Kolodny EH, Pastores GM, Muenzer J, Rapoport DM, Berger KI, Swiedler SJ, Kakkis ED, Braakman T, Chadbourne E, Walton-Bowen K, Cox GF. PMID: 15126990.
      View in: PubMed   Mentions: 186     Fields:    Translation:HumansCTClinical Trials
    52. Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I. Proc Natl Acad Sci U S A. 2004 Jan 20; 101(3):829-34. Kakkis E, Lester T, Yang R, Tanaka C, Anand V, Lemontt J, Peinovich M, Passage M. PMID: 14715900; PMCID: PMC321766.
      View in: PubMed   Mentions: 26     Fields:    Translation:HumansAnimals
    53. Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I. Lancet. 2003 May 10; 361(9369):1608-13. Kakavanos R, Turner CT, Hopwood JJ, Kakkis ED, Brooks DA. PMID: 12747881.
      View in: PubMed   Mentions: 31     Fields:    Translation:HumansAnimals
    54. Enzyme replacement therapy for the mucopolysaccharide storage disorders. Expert Opin Investig Drugs. 2002 May; 11(5):675-85. Kakkis ED. PMID: 11996648.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansAnimals
    55. Enzyme replacement therapy in feline mucopolysaccharidosis I. Mol Genet Metab. 2001 Mar; 72(3):199-208. Kakkis ED, Schuchman E, He X, Wan Q, Kania S, Wiemelt S, Hasson CW, O'Malley T, Weil MA, Aguirre GA, Brown DE, Haskins ME. PMID: 11243725.
      View in: PubMed   Mentions: 28     Fields:    Translation:Animals
    56. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med. 2001 Jan 18; 344(3):182-8. Kakkis ED, Muenzer J, Tiller GE, Waber L, Belmont J, Passage M, Izykowski B, Phillips J, Doroshow R, Walot I, Hoft R, Neufeld EF. PMID: 11172140.
      View in: PubMed   Mentions: 212     Fields:    Translation:HumansCTClinical Trials
    57. Retrovirus-mediated transfer of the human alpha-L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts. Gene Ther. 1997 Nov; 4(11):1150-9. Huang MM, Wong A, Yu X, Kakkis E, Kohn DB. PMID: 9425437.
      View in: PubMed   Mentions: 4     Fields:    Translation:AnimalsCells
    58. Carbohydrate structures of recombinant human alpha-L-iduronidase secreted by Chinese hamster ovary cells. J Biol Chem. 1997 Sep 05; 272(36):22758-65. Zhao KW, Faull KF, Kakkis ED, Neufeld EF. PMID: 9278435.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansAnimalsCells
    59. Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I. Biochem Mol Med. 1996 Aug; 58(2):156-67. Kakkis ED, McEntee MF, Schmidtchen A, Neufeld EF, Ward DA, Gompf RE, Kania S, Bedolla C, Chien SL, Shull RM. PMID: 8812735.
      View in: PubMed   Mentions: 46     Fields:    Translation:HumansAnimalsCells
    60. Enzyme replacement in a canine model of Hurler syndrome. Proc Natl Acad Sci U S A. 1994 Dec 20; 91(26):12937-41. Shull RM, Kakkis ED, McEntee MF, Kania SA, Jonas AJ, Neufeld EF. PMID: 7809150; PMCID: PMC45555.
      View in: PubMed   Mentions: 44     Fields:    Translation:Animals
    61. Acute lymphocytic leukemia in acrodermatitis enteropathica. Am J Hematol. 1994 Aug; 46(4):374-5. Lin HJ, Kakkis ED, Sun NC, Lim S, Gurevitch AW. PMID: 8037198.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    62. Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells. Protein Expr Purif. 1994 Jun; 5(3):225-32. Kakkis ED, Matynia A, Jonas AJ, Neufeld EF. PMID: 7950365.
      View in: PubMed   Mentions: 29     Fields:    Translation:HumansAnimalsCells
    63. DOOR syndrome (deafness, onycho-osteodystrophy, and mental retardation): a new patient and delineation of neurologic variability among recessive cases. Am J Med Genet. 1993 Sep 15; 47(4):534-9. Lin HJ, Kakkis ED, Eteson DJ, Lachman RS. PMID: 8256819.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    64. A transcriptional repressor of c-myc. Nature. 1989 Jun 29; 339(6227):718-21. Kakkis E, Riggs KJ, Gillespie W, Calame K. PMID: 2662014.
      View in: PubMed   Mentions: 34     Fields:    Translation:HumansAnimalsCells
    65. Strong transcriptional activation of translocated c-myc genes occurs without a strong nearby enhancer or promoter. Nucleic Acids Res. 1988 Jan 11; 16(1):77-96. Kakkis E, Mercola M, Calame K. PMID: 2829126; PMCID: PMC334614.
      View in: PubMed   Mentions: 5     Fields:    Translation:AnimalsCells
    66. A repressor of c-myc transcription is found specifically in plasmacytomas. Curr Top Microbiol Immunol. 1988; 141:231-7. Kakkis E, Riggs K, Calame K. PMID: 3215052.
      View in: PubMed   Mentions:    Fields:    Translation:AnimalsCells
    67. Transcription factors in terminally differentiated B cells. Curr Top Microbiol Immunol. 1988; 137:100-6. Peterson CL, Tsao B, Kakkis E, Calame KL. PMID: 3138069.
      View in: PubMed   Mentions:    Fields:    Translation:AnimalsCells
    68. A plasmacytoma-specific factor binds the c-myc promoter region. Proc Natl Acad Sci U S A. 1987 Oct; 84(20):7031-5. Kakkis E, Calame K. PMID: 3313385; PMCID: PMC299223.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansAnimalsCells
    69. An active chromatin structure acquired by translocated c-myc genes. Mol Cell Biol. 1986 Apr; 6(4):1357-61. Kakkis E, Prehn J, Calame K. PMID: 3023885; PMCID: PMC367653.
      View in: PubMed   Mentions: 7     Fields:    Translation:AnimalsCells
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