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Results: 1 to 20 of 97

1.

Pharmacological chaperones as therapeutics for lysosomal storage diseases.

Boyd RE, Lee G, Rybczynski P, Benjamin ER, Khanna R, Wustman BA, Valenzano KJ.

J Med Chem. 2013 Apr 11;56(7):2705-25. doi: 10.1021/jm301557k. Epub 2013 Mar 11. Review.

PMID:
23363020
[PubMed - indexed for MEDLINE]
2.

Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders.

Valenzano KJ, Khanna R, Powe AC, Boyd R, Lee G, Flanagan JJ, Benjamin ER.

Assay Drug Dev Technol. 2011 Jun;9(3):213-35. doi: 10.1089/adt.2011.0370. Review.

PMID:
21612550
[PubMed - indexed for MEDLINE]
Free PMC Article
3.

Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics.

Parenti G.

EMBO Mol Med. 2009 Aug;1(5):268-79. doi: 10.1002/emmm.200900036. Review.

PMID:
20049730
[PubMed - indexed for MEDLINE]
Free PMC Article
4.

Therapeutic strategies to ameliorate lysosomal storage disorders--a focus on Gaucher disease.

Sawkar AR, D'Haeze W, Kelly JW.

Cell Mol Life Sci. 2006 May;63(10):1179-92. Review.

PMID:
16568247
[PubMed - indexed for MEDLINE]
5.

A chaperone-mediated approach to enzyme enhancement as a therapeutic option for the lysosomal storage disorders.

Pastores GM, Sathe S.

Drugs R D. 2006;7(6):339-48. Review.

PMID:
17073517
[PubMed - indexed for MEDLINE]
6.

A counterintuitive approach to treat enzyme deficiencies: use of enzyme inhibitors for restoring mutant enzyme activity.

Fan JQ.

Biol Chem. 2008 Jan;389(1):1-11. Review.

PMID:
18095864
[PubMed - indexed for MEDLINE]
7.

Pharmacological chaperones for enzyme enhancement therapy in genetic diseases.

Aymami J, Barril X, Rodríguez-Pascau L, Martinell M.

Pharm Pat Anal. 2013 Jan;2(1):109-24. doi: 10.4155/ppa.12.74. Review.

PMID:
24236974
[PubMed - indexed for MEDLINE]
8.

Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.

Butters TD.

Expert Opin Pharmacother. 2007 Mar;8(4):427-35. Review.

PMID:
17309337
[PubMed - indexed for MEDLINE]
9.

Recent developments in therapeutic approaches for lysosomal storage diseases.

Urbanelli L, Magini A, Polchi A, Polidoro M, Emiliani C.

Recent Pat CNS Drug Discov. 2011 Jan;6(1):1-19. Review.

PMID:
21073432
[PubMed - indexed for MEDLINE]
10.

Cyclodextrin-based iminosugar click clusters: the first examples of multivalent pharmacological chaperones for the treatment of lysosomal storage disorders.

Decroocq C, Rodríguez-Lucena D, Ikeda K, Asano N, Compain P.

Chembiochem. 2012 Mar 19;13(5):661-4. doi: 10.1002/cbic.201200005. Epub 2012 Feb 16. No abstract available.

PMID:
22344719
[PubMed - indexed for MEDLINE]
11.

Chemical and biological approaches synergize to ameliorate protein-folding diseases.

Mu TW, Ong DS, Wang YJ, Balch WE, Yates JR 3rd, Segatori L, Kelly JW.

Cell. 2008 Sep 5;134(5):769-81. doi: 10.1016/j.cell.2008.06.037.

PMID:
18775310
[PubMed - indexed for MEDLINE]
Free PMC Article
12.

Therapeutic applications of imino sugars in lysosomal storage disorders.

Butters TD, Dwek RA, Platt FM.

Curr Top Med Chem. 2003;3(5):561-74. Review.

PMID:
12570866
[PubMed - indexed for MEDLINE]
13.

Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.

Desnick RJ, Schuchman EH.

Nat Rev Genet. 2002 Dec;3(12):954-66. Review. Erratum in: Nat Rev Genet. 2003 Feb;4(2):157..

PMID:
12459725
[PubMed - indexed for MEDLINE]
14.

Pharmacological chaperone corrects lysosomal storage in Fabry disease caused by trafficking-incompetent variants.

Yam GH, Bosshard N, Zuber C, Steinmann B, Roth J.

Am J Physiol Cell Physiol. 2006 Apr;290(4):C1076-82.

PMID:
16531566
[PubMed - indexed for MEDLINE]
Free Article
15.

Partial restoration of mutant enzyme homeostasis in three distinct lysosomal storage disease cell lines by altering calcium homeostasis.

Mu TW, Fowler DM, Kelly JW.

PLoS Biol. 2008 Feb;6(2):e26. doi: 10.1371/journal.pbio.0060026.

PMID:
18254660
[PubMed - indexed for MEDLINE]
Free PMC Article
16.

Enzyme replacement and enhancement therapies for lysosomal diseases.

Desnick RJ.

J Inherit Metab Dis. 2004;27(3):385-410. Review.

PMID:
15190196
[PubMed - indexed for MEDLINE]
17.

[Metabolic pathway of the degradation of macromolecules by lysosomal enzymes].

Arai K, Ohkuma S.

Nihon Rinsho. 1995 Dec;53(12):2904-10. Review. Japanese.

PMID:
8577033
[PubMed - indexed for MEDLINE]
18.

[Therapy of lysosomal storage diseases: update and perspectives].

Lara-Aguilar RA, Juárez-Vázquez CI, Medina-Lozano C.

Rev Invest Clin. 2011 Nov-Dec;63(6):651-8. Review. Spanish.

PMID:
23650678
[PubMed - indexed for MEDLINE]
19.

Pharmacological small molecules for the treatment of lysosomal storage disorders.

Smid BE, Aerts JM, Boot RG, Linthorst GE, Hollak CE.

Expert Opin Investig Drugs. 2010 Nov;19(11):1367-79. doi: 10.1517/13543784.2010.524205. Epub 2010 Oct 13. Review. Erratum in: Expert Opin Investig Drugs. 2011 Feb;20(2):309.

PMID:
20942596
[PubMed - indexed for MEDLINE]
20.

Update on treatment of lysosomal storage diseases.

Bruni S, Loschi L, Incerti C, Gabrielli O, Coppa GV.

Acta Myol. 2007 Jul;26(1):87-92. Review.

PMID:
17915580
[PubMed - indexed for MEDLINE]
Free PMC Article

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