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Items: 1 to 20 of 105

1.

Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study.

Cox TM, Amato D, Hollak CE, Luzy C, Silkey M, Giorgino R, Steiner RD; Miglustat Maintenance Study Group.

Orphanet J Rare Dis. 2012 Dec 27;7:102. doi: 10.1186/1750-1172-7-102.

2.
3.

Enzyme replacement and substrate reduction therapy for Gaucher disease.

Shemesh E, Deroma L, Bembi B, Deegan P, Hollak C, Weinreb NJ, Cox TM.

Cochrane Database Syst Rev. 2015 Mar 27;(3):CD010324. doi: 10.1002/14651858.CD010324.pub2. Review.

PMID:
25812601
4.

Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project.

Giraldo P, Alfonso P, Atutxa K, Fernández-Galán MA, Barez A, Franco R, Alonso D, Martin A, Latre P, Pocovi M.

Haematologica. 2009 Dec;94(12):1771-5. doi: 10.3324/haematol.2009.008078. Epub 2009 Jul 16.

5.

Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.

Elstein D, Dweck A, Attias D, Hadas-Halpern I, Zevin S, Altarescu G, Aerts JF, van Weely S, Zimran A.

Blood. 2007 Oct 1;110(7):2296-301. Epub 2007 Jul 3.

6.

Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease.

Elstein D, Hollak C, Aerts JM, van Weely S, Maas M, Cox TM, Lachmann RH, Hrebicek M, Platt FM, Butters TD, Dwek RA, Zimran A.

J Inherit Metab Dis. 2004;27(6):757-66.

PMID:
15505381
7.

Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report.

Giuffrida G, Lombardo R, Di Francesco E, Parrinello L, Di Raimondo F, Fiumara A.

J Med Case Rep. 2016 Nov 8;10(1):315.

8.

Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme.

Hollak CE, Hughes D, van Schaik IN, Schwierin B, Bembi B.

Pharmacoepidemiol Drug Saf. 2009 Sep;18(9):770-7. doi: 10.1002/pds.1779.

PMID:
19507165
9.
10.

Miglustat therapy in type 1 Gaucher disease: clinical and safety outcomes in a multicenter retrospective cohort study.

Kuter DJ, Mehta A, Hollak CE, Giraldo P, Hughes D, Belmatoug N, Brand M, Muller A, Schaaf B, Giorgino R, Zimran A.

Blood Cells Mol Dis. 2013 Aug;51(2):116-24. doi: 10.1016/j.bcmd.2013.04.005. Epub 2013 May 14.

PMID:
23683771
11.

Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series.

Serratrice C, Swiader L, Serratrice J.

J Med Case Rep. 2015 Jun 23;9:146. doi: 10.1186/s13256-015-0617-5.

12.

Results from a 9-year Intensive Safety Surveillance Scheme (IS(3) ) in miglustat (Zavesca(®) )-treated patients.

Brand M, Muller A, Alsop J, van Schaik IN, Bembi B, Hughes D.

Pharmacoepidemiol Drug Saf. 2015 Mar;24(3):329-33. doi: 10.1002/pds.3760. Epub 2015 Feb 5.

PMID:
25656910
13.

Substrate reduction therapy with miglustat for type 1 Gaucher disease: a retrospective analysis from a single institution.

Machaczka M, Hast R, Dahlman I, Lerner R, Klimkowska M, Engvall M, Hägglund H.

Ups J Med Sci. 2012 Mar;117(1):28-34. doi: 10.3109/03009734.2011.641609. Epub 2012 Jan 17.

14.

A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustat.

Remenova T, Morand O, Amato D, Chadha-Boreham H, Tsurutani S, Marquardt T.

Orphanet J Rare Dis. 2015 Jun 19;10:81. doi: 10.1186/s13023-015-0297-7.

15.

Randomized, controlled trial of miglustat in Gaucher's disease type 3.

Schiffmann R, Fitzgibbon EJ, Harris C, DeVile C, Davies EH, Abel L, van Schaik IN, Benko W, Timmons M, Ries M, Vellodi A.

Ann Neurol. 2008 Nov;64(5):514-22. doi: 10.1002/ana.21491.

16.

Computerized cognitive testing in patients with type I Gaucher disease: effects of enzyme replacement and substrate reduction.

Elstein D, Guedalia J, Doniger GM, Simon ES, Antebi V, Arnon Y, Zimran A.

Genet Med. 2005 Feb;7(2):124-30.

PMID:
15714080
17.

Gastrointestinal disturbances and their management in miglustat-treated patients.

Belmatoug N, Burlina A, Giraldo P, Hendriksz CJ, Kuter DJ, Mengel E, Pastores GM.

J Inherit Metab Dis. 2011 Oct;34(5):991-1001. doi: 10.1007/s10545-011-9368-7. Epub 2011 Jul 21. Review.

PMID:
21779792
18.

Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment.

Shapiro BE, Pastores GM, Gianutsos J, Luzy C, Kolodny EH.

Genet Med. 2009 Jun;11(6):425-33. doi: 10.1097/GIM.0b013e3181a1b5c5.

PMID:
19346952
19.

Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial.

Mistry PK, Lukina E, Ben Turkia H, Amato D, Baris H, Dasouki M, Ghosn M, Mehta A, Packman S, Pastores G, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Shankar S, Solano MH, Ross L, Angell J, Peterschmitt MJ.

JAMA. 2015 Feb 17;313(7):695-706. doi: 10.1001/jama.2015.459.

20.

Goal-oriented therapy with miglustat in Gaucher disease.

Pastores GM, Giraldo P, Chérin P, Mehta A.

Curr Med Res Opin. 2009 Jan;25(1):23-37. doi: 10.1185/03007990802576518 . Review.

PMID:
19210136

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