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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2002 7
2003 6
2004 14
2005 5
2006 4
2007 9
2008 12
2009 5
2010 8
2011 5
2012 4
2013 7
2014 9
2015 13
2016 12
2017 11
2018 10
2019 10
2020 11
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Search Results

143 results
Results by year
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Page 1
Lysosomal storage diseases.
Platt FM, d'Azzo A, Davidson BL, Neufeld EF, Tifft CJ. Platt FM, et al. Nat Rev Dis Primers. 2018 Oct 1;4(1):27. doi: 10.1038/s41572-018-0025-4. Nat Rev Dis Primers. 2018. PMID: 30275469 Review.
Fetal gene therapy for neurodegenerative disease of infants.
Massaro G, Mattar CNZ, Wong AMS, Sirka E, Buckley SMK, Herbert BR, Karlsson S, Perocheau DP, Burke D, Heales S, Richard-Londt A, Brandner S, Huebecker M, Priestman DA, Platt FM, Mills K, Biswas A, Cooper JD, Chan JKY, Cheng SH, Waddington SN, Rahim AA. Massaro G, et al. Among authors: platt fm. Nat Med. 2018 Sep;24(9):1317-1323. doi: 10.1038/s41591-018-0106-7. Epub 2018 Jul 16. Nat Med. 2018. PMID: 30013199 Free PMC article.
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.
Emptying the stores: lysosomal diseases and therapeutic strategies.
Platt FM. Platt FM. Nat Rev Drug Discov. 2018 Feb;17(2):133-150. doi: 10.1038/nrd.2017.214. Epub 2017 Nov 17. Nat Rev Drug Discov. 2018. PMID: 29147032 Review.
Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses.
Kirkegaard T, Gray J, Priestman DA, Wallom KL, Atkins J, Olsen OD, Klein A, Drndarski S, Petersen NH, Ingemann L, Smith DA, Morris L, Bornæs C, Jørgensen SH, Williams I, Hinsby A, Arenz C, Begley D, Jäättelä M, Platt FM. Kirkegaard T, et al. Among authors: platt fm. Sci Transl Med. 2016 Sep 7;8(355):355ra118. doi: 10.1126/scitranslmed.aad9823. Sci Transl Med. 2016. PMID: 27605553 Free PMC article.
AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease.
Hughes MP, Smith DA, Morris L, Fletcher C, Colaco A, Huebecker M, Tordo J, Palomar N, Massaro G, Henckaerts E, Waddington SN, Platt FM, Rahim AA. Hughes MP, et al. Among authors: platt fm. Hum Mol Genet. 2018 Sep 1;27(17):3079-3098. doi: 10.1093/hmg/ddy212. Hum Mol Genet. 2018. PMID: 29878115 Free PMC article.
Reduced sphingolipid hydrolase activities, substrate accumulation and ganglioside decline in Parkinson's disease.
Huebecker M, Moloney EB, van der Spoel AC, Priestman DA, Isacson O, Hallett PJ, Platt FM. Huebecker M, et al. Among authors: platt fm. Mol Neurodegener. 2019 Nov 8;14(1):40. doi: 10.1186/s13024-019-0339-z. Mol Neurodegener. 2019. PMID: 31703585 Free PMC article.
Mechanistic convergence and shared therapeutic targets in Niemann-Pick disease.
Colaco A, Kaya E, Adriaenssens E, Davis LC, Zampieri S, Fernández-Suárez ME, Tan CY, Deegan PB, Porter FD, Galione A, Bembi B, Dardis A, Platt FM. Colaco A, et al. Among authors: platt fm. J Inherit Metab Dis. 2020 May;43(3):574-585. doi: 10.1002/jimd.12191. Epub 2019 Dec 5. J Inherit Metab Dis. 2020. PMID: 31707734 Free PMC article.
Publisher Correction: Lysosomal storage diseases.
Platt FM, d'Azzo A, Davidson BL, Neufeld EF, Tifft CJ. Platt FM, et al. Nat Rev Dis Primers. 2019 May 17;5(1):34. doi: 10.1038/s41572-019-0089-9. Nat Rev Dis Primers. 2019. PMID: 31101820
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