Kakkis E - Search Results

1

Intrathecal administration of recombinant human N-acetylgalactosamine 4-sulfatase to a MPS VI patient with pachymeningitis cervicalis.

Muñoz-Rojas MV, Horovitz DD, Jardim LB, Raymundo M, Llerena JC Jr, de Magalhães Tde S, Vieira TA, Costa R, Kakkis E, Giugliani R. Muñoz-Rojas MV, et al. Among authors: kakkis e. Mol Genet Metab. 2010 Apr;99(4):346-50. doi: 10.1016/j.ymgme.2009.11.008. Epub 2009 Dec 5. Mol Genet Metab. 2010. PMID: 20036175

2

Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression.

Munoz-Rojas MV, Vieira T, Costa R, Fagondes S, John A, Jardim LB, Vedolin LM, Raymundo M, Dickson PI, Kakkis E, Giugliani R. Munoz-Rojas MV, et al. Among authors: kakkis e. Am J Med Genet A. 2008 Oct 1;146A(19):2538-44. doi: 10.1002/ajmg.a.32294. Am J Med Genet A. 2008. PMID: 18792977

3

A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I.

Giugliani R, Rojas VM, Martins AM, Valadares ER, Clarke JT, Góes JE, Kakkis ED, Worden MA, Sidman M, Cox GF. Giugliani R, et al. Among authors: kakkis ed. Mol Genet Metab. 2009 Jan;96(1):13-9. doi: 10.1016/j.ymgme.2008.10.009. Epub 2008 Nov 26. Mol Genet Metab. 2009. PMID: 19038563 Clinical Trial.

4

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.

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. Harmatz P, et al. Among authors: kakkis e. J Pediatr. 2006 Apr;148(4):533-539. doi: 10.1016/j.jpeds.2005.12.014. J Pediatr. 2006. PMID: 16647419 Clinical Trial.

5

Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen.

Auclair D, Finnie J, White J, Nielsen T, Fuller M, Kakkis E, Cheng A, O'Neill CA, Hopwood JJ. Auclair D, et al. Among authors: kakkis e. Mol Genet Metab. 2010 Feb;99(2):132-41. doi: 10.1016/j.ymgme.2009.10.002. Epub 2009 Oct 13. Mol Genet Metab. 2010. PMID: 19896877

6

Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I.

Kakkis E, McEntee M, Vogler C, Le S, Levy B, Belichenko P, Mobley W, Dickson P, Hanson S, Passage M. Kakkis E, et al. Mol Genet Metab. 2004 Sep-Oct;83(1-2):163-74. doi: 10.1016/j.ymgme.2004.07.003. Mol Genet Metab. 2004. PMID: 15464431

7

Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid.

Dickson P, McEntee M, Vogler C, Le S, Levy B, Peinovich M, Hanson S, Passage M, Kakkis E. Dickson P, et al. Among authors: kakkis e. Mol Genet Metab. 2007 May;91(1):61-8. doi: 10.1016/j.ymgme.2006.12.012. Epub 2007 Feb 26. Mol Genet Metab. 2007. PMID: 17321776 Free PMC article.

8

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

Cadaoas J, Boyle G, Jungles S, Cullen S, Vellard M, Grubb JH, Jurecka A, Sly W, Kakkis E. Cadaoas J, et al. Among authors: kakkis e. Mol Genet Metab. 2020 May;130(1):65-76. doi: 10.1016/j.ymgme.2020.02.009. Epub 2020 Mar 6. Mol Genet Metab. 2020. PMID: 32192868 Free article. Clinical Trial.

9

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

Fox JE, Volpe L, Bullaro J, Kakkis ED, Sly WS. Fox JE, et al. Among authors: kakkis ed. Mol Genet Metab. 2015 Feb;114(2):203-8. doi: 10.1016/j.ymgme.2014.10.017. Epub 2014 Nov 7. Mol Genet Metab. 2015. PMID: 25468648 Free PMC article.

10

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

Jones SA, Marsden D, Koutsoukos T, Sniadecki J, Tylee K, Phillippo S, Kakkis E. Jones SA, et al. Among authors: kakkis e. Mol Genet Metab. 2020 Aug;130(4):255-261. doi: 10.1016/j.ymgme.2020.06.004. Epub 2020 Jun 11. Mol Genet Metab. 2020. PMID: 32563631 Free article.

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