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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 14
2021 11
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156 results
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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.
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.
c-Abl Inhibition Activates TFEB and Promotes Cellular Clearance in a Lysosomal Disorder.
Contreras PS, Tapia PJ, González-Hódar L, Peluso I, Soldati C, Napolitano G, Matarese M, Heras ML, Valls C, Martinez A, Balboa E, Castro J, Leal N, Platt FM, Sobota A, Winter D, Klein AD, Medina DL, Ballabio A, Alvarez AR, Zanlungo S. Contreras PS, et al. Among authors: platt fm. iScience. 2020 Oct 15;23(11):101691. doi: 10.1016/j.isci.2020.101691. eCollection 2020 Nov 20. iScience. 2020. PMID: 33163944 Free PMC article.
Defective platelet function in Niemann-Pick disease type C1.
Chen OCW, Colaco A, Davis LC, Kiskin FN, Farhat NY, Speak AO, Smith DA, Morris L, Eden E, Tynan P, Churchill GC, Galione A, Porter FD, Platt FM. Chen OCW, et al. Among authors: platt fm. JIMD Rep. 2020 Sep 12;56(1):46-57. doi: 10.1002/jmd2.12148. eCollection 2020 Nov. JIMD Rep. 2020. PMID: 33204596 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.
Substrate reduction therapy.
Platt FM, Jeyakumar M. Platt FM, et al. Acta Paediatr. 2008 Apr;97(457):88-93. doi: 10.1111/j.1651-2227.2008.00656.x. Acta Paediatr. 2008. PMID: 18339196 Review.
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