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Sphingolipid lysosomal storage disorders.
Platt FM. Platt FM. Nature. 2014 Jun 5;510(7503):68-75. doi: 10.1038/nature13476. Nature. 2014. PMID: 24899306 Review.
N-butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis.
Platt FM, Neises GR, Dwek RA, Butters TD. Platt FM, et al. J Biol Chem. 1994 Mar 18;269(11):8362-5. J Biol Chem. 1994. PMID: 8132559
Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin.
Platt FM, Neises GR, Reinkensmeier G, Townsend MJ, Perry VH, Proia RL, Winchester B, Dwek RA, Butters TD. Platt FM, et al. Science. 1997 Apr 18;276(5311):428-31. doi: 10.1126/science.276.5311.428. Science. 1997. PMID: 9103204
New therapeutic prospects for the glycosphingolipid lysosomal storage diseases.
Platt FM, Butters TD. Platt FM, et al. Biochem Pharmacol. 1998 Aug 15;56(4):421-30. doi: 10.1016/s0006-2952(98)00115-4. Biochem Pharmacol. 1998. PMID: 9763217 Review.
Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin.
Jeyakumar M, Butters TD, Cortina-Borja M, Hunnam V, Proia RL, Perry VH, Dwek RA, Platt FM. Jeyakumar M, et al. Among authors: platt fm. Proc Natl Acad Sci U S A. 1999 May 25;96(11):6388-93. doi: 10.1073/pnas.96.11.6388. Proc Natl Acad Sci U S A. 1999. PMID: 10339597 Free PMC article.
N-butyldeoxygalactonojirimycin: a more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivo.
Andersson U, Butters TD, Dwek RA, Platt FM. Andersson U, et al. Among authors: platt fm. Biochem Pharmacol. 2000 Apr 1;59(7):821-9. doi: 10.1016/s0006-2952(99)00384-6. Biochem Pharmacol. 2000. PMID: 10718340
High-performance cation-exchange chromatography and pulsed amperometric detection for the separation, detection, and quantitation of N-alkylated imino sugars in biological samples.
Mellor HR, Adam A, Platt FM, Dwek RA, Butters TD. Mellor HR, et al. Among authors: platt fm. Anal Biochem. 2000 Aug 15;284(1):136-42. doi: 10.1006/abio.2000.4678. Anal Biochem. 2000. PMID: 10933866
Substrate reduction therapy for glycosphingolipid storage disorders.
Lachmann RH, Platt FM. Lachmann RH, et al. Among authors: platt fm. Expert Opin Investig Drugs. 2001 Mar;10(3):455-66. doi: 10.1517/13543784.10.3.455. Expert Opin Investig Drugs. 2001. PMID: 11227045 Review.
Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy.
Platt FM, Jeyakumar M, Andersson U, Priestman DA, Dwek RA, Butters TD, Cox TM, Lachmann RH, Hollak C, Aerts JM, Van Weely S, HrebĂ­cek M, Moyses C, Gow I, Elstein D, Zimran A. Platt FM, et al. J Inherit Metab Dis. 2001 Apr;24(2):275-90. doi: 10.1023/a:1010335505357. J Inherit Metab Dis. 2001. PMID: 11405346 Review.
Inhibition of glycosphingolipid biosynthesis: application to lysosomal storage disorders.
Butters TD, Dwek RA, Platt FM. Butters TD, et al. Among authors: platt fm. Chem Rev. 2000 Dec 13;100(12):4683-96. doi: 10.1021/cr990292q. Chem Rev. 2000. PMID: 11749362 No abstract available.
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