Kakkis ED - Search Results

1

The transformation of drug development for the 21st century: Time for a change.

Kakkis ED. Kakkis ED. Mol Genet Metab. 2022 Sep-Oct;137(1-2):107-113. doi: 10.1016/j.ymgme.2022.07.015. Epub 2022 Aug 2. Mol Genet Metab. 2022. PMID: 35985171 Free article. No abstract available.

2

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

Kakkis E, Marsden D. Kakkis E, et al. Mol Genet Metab. 2020 May;130(1):7-15. doi: 10.1016/j.ymgme.2020.02.006. Epub 2020 Feb 19. Mol Genet Metab. 2020. PMID: 32224020 Free article. Review.

3

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. 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.

4

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. 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.

5

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 J, Marsden D, McCracken E, DeWard S, Barone A, Hsu K, Kakkis E. Vockley J, et al. Mol Genet Metab. 2015 Sep-Oct;116(1-2):53-60. doi: 10.1016/j.ymgme.2015.06.006. Epub 2015 Jun 18. Mol Genet Metab. 2015. PMID: 26116311 Free PMC article. Review.

6

A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease.

Harmatz P, Whitley CB, Wang RY, Bauer M, Song W, Haller C, Kakkis E. Harmatz P, et al. Mol Genet Metab. 2018 Apr;123(4):488-494. doi: 10.1016/j.ymgme.2018.02.006. Epub 2018 Feb 12. Mol Genet Metab. 2018. PMID: 29478819 Free article. Clinical Trial.

7

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.

8

UX007 for the treatment of long chain-fatty acid oxidation disorders: Safety and efficacy in children and adults following 24weeks of treatment.

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. Vockley J, et al. Mol Genet Metab. 2017 Apr;120(4):370-377. doi: 10.1016/j.ymgme.2017.02.005. Epub 2017 Feb 7. Mol Genet Metab. 2017. PMID: 28189603 Free article. Clinical Trial.

9

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

10

Enzyme replacement therapy in feline mucopolysaccharidosis I.

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. Kakkis ED, et al. Mol Genet Metab. 2001 Mar;72(3):199-208. doi: 10.1006/mgme.2000.3140. Mol Genet Metab. 2001. PMID: 11243725

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