Emil Kakkis[Author] - Search Results

Year	Number of Results
2002	1
2003	1
2004	1
2005	2
2006	2
2007	3
2008	3
2009	5
2010	3
2011	3
2012	2
2013	1
2014	1
2015	4
2016	3
2017	2
2018	3
2019	2
2020	5
2021	4
2022	2
2023	1
2024	1

1

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.

2

Enzyme replacement therapy for the mucopolysaccharide storage disorders.

Kakkis ED. Kakkis ED. Expert Opin Investig Drugs. 2002 May;11(5):675-85. doi: 10.1517/13543784.11.5.675. Expert Opin Investig Drugs. 2002. PMID: 11996648 Review.

3

Burosumab Therapy in Children with X-Linked Hypophosphatemia.

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. Carpenter TO, et al. Among authors: kakkis e. N Engl J Med. 2018 May 24;378(21):1987-1998. doi: 10.1056/NEJMoa1714641. N Engl J Med. 2018. PMID: 29791829 Free article. Clinical Trial.

4

The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases.

Tandon PK, Kakkis ED. Tandon PK, et al. Among authors: kakkis ed. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5. Orphanet J Rare Dis. 2021. PMID: 33874971 Free PMC article. Review.

5

The 21st Century Cures Act.

Kakkis E, Bronstein MG. Kakkis E, et al. N Engl J Med. 2015 Oct 22;373(17):1678. doi: 10.1056/NEJMc1509640. N Engl J Med. 2015. PMID: 26488712 No abstract available.

6

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.

7

Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I.

Kakavanos R, Turner CT, Hopwood JJ, Kakkis ED, Brooks DA. Kakavanos R, et al. Among authors: kakkis ed. Lancet. 2003 May 10;361(9369):1608-13. doi: 10.1016/S0140-6736(03)13311-9. Lancet. 2003. PMID: 12747881 Review.

8

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. Among authors: kakkis e. 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.

9

Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints.

Kakkis ED, O'Donovan M, Cox G, Hayes M, Goodsaid F, Tandon PK, Furlong P, Boynton S, Bozic M, Orfali M, Thornton M. Kakkis ED, et al. Orphanet J Rare Dis. 2015 Feb 10;10:16. doi: 10.1186/s13023-014-0195-4. Orphanet J Rare Dis. 2015. PMID: 25757705 Free PMC article. Review.

10

Conventional and Unconventional Therapeutic Strategies for Sialidosis Type I.

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. Mosca R, et al. Among authors: kakkis e. J Clin Med. 2020 Mar 4;9(3):695. doi: 10.3390/jcm9030695. J Clin Med. 2020. PMID: 32143456 Free PMC article.

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