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A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.
Lukina E, Watman N, Arreguin EA, Banikazemi M, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Rosenthal DI, Kaper M, Singh T, Puga AC, Bonate PL, Peterschmitt MJ. Lukina E, et al. Among authors: phillips m. Blood. 2010 Aug 12;116(6):893-9. doi: 10.1182/blood-2010-03-273151. Epub 2010 May 3. Blood. 2010. PMID: 20439622 Free PMC article. Clinical Trial.
Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study.
Lukina E, Watman N, Arreguin EA, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Kamath RS, Rosenthal DI, Kaper M, Singh T, Puga AC, Peterschmitt MJ. Lukina E, et al. Among authors: phillips m. Blood. 2010 Nov 18;116(20):4095-8. doi: 10.1182/blood-2010-06-293902. Epub 2010 Aug 16. Blood. 2010. PMID: 20713962 Free PMC article. Clinical Trial.
Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease.
Zimran A, Brill-Almon E, Chertkoff R, Petakov M, Blanco-Favela F, Muñoz ET, Solorio-Meza SE, Amato D, Duran G, Giona F, Heitner R, Rosenbaum H, Giraldo P, Mehta A, Park G, Phillips M, Elstein D, Altarescu G, Szleifer M, Hashmueli S, Aviezer D. Zimran A, et al. Among authors: phillips m. Blood. 2011 Nov 24;118(22):5767-73. doi: 10.1182/blood-2011-07-366955. Epub 2011 Sep 6. Blood. 2011. PMID: 21900191 Free article. Clinical Trial.
Phenotype in patients with Gaucher disease and Parkinson disease.
Chetrit EB, Alcalay RN, Steiner-Birmanns B, Altarescu G, Phillips M, Elstein D, Zimran A. Chetrit EB, et al. Among authors: phillips m. Blood Cells Mol Dis. 2013 Mar;50(3):218-21. doi: 10.1016/j.bcmd.2012.11.011. Epub 2012 Dec 20. Blood Cells Mol Dis. 2013. PMID: 23265741
Disease severity in sibling pairs with type 1 Gaucher disease.
Elstein D, Gellman A, Altarescu G, Abrahamov A, Hadas-Halpern I, Phillips M, Margalit M, Lebel E, Itzchaki M, Zimran A. Elstein D, et al. Among authors: phillips m. J Inherit Metab Dis. 2010 Feb;33(1):79-83. doi: 10.1007/s10545-009-9024-7. Epub 2010 Jan 5. J Inherit Metab Dis. 2010. PMID: 20049528
4,953 results