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Journal Abstract Search
600 related items for PubMed ID: 17689147
1. Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE. Lancet Neurol; 2007 Sep; 6(9):765-72. PubMed ID: 17689147 [Abstract] [Full Text] [Related]
2. Long-term miglustat therapy in children with Niemann-Pick disease type C. Patterson MC, Vecchio D, Jacklin E, Abel L, Chadha-Boreham H, Luzy C, Giorgino R, Wraith JE. J Child Neurol; 2010 Mar; 25(3):300-5. PubMed ID: 19822772 [Abstract] [Full Text] [Related]
5. Treatment of Niemann-Pick disease type C in two children with miglustat: initial responses and maintenance of effects over 1 year. Chien YH, Lee NC, Tsai LK, Huang AC, Peng SF, Chen SJ, Hwu WL. J Inherit Metab Dis; 2007 Oct; 30(5):826. PubMed ID: 17603755 [Abstract] [Full Text] [Related]
6. Efficacy of miglustat in Niemann-Pick C disease: a single centre experience. Ginocchio VM, D'Amico A, Bertini E, Ceravolo F, Dardis A, Verrigni D, Bembi B, Dionisi-Vici C, Deodato F. Mol Genet Metab; 2013 Nov; 110(3):329-35. PubMed ID: 23973268 [Abstract] [Full Text] [Related]
7. Longitudinal assessment of reflexive and volitional saccades in Niemann-Pick Type C disease during treatment with miglustat. Abel LA, Walterfang M, Stainer MJ, Bowman EA, Velakoulis D. Orphanet J Rare Dis; 2015 Dec 21; 10():160. PubMed ID: 26691656 [Abstract] [Full Text] [Related]
10. Treatment of a child diagnosed with Niemann-Pick disease type C with miglustat: a case report in Brazil. Santos ML, Raskin S, Telles DS, Löhr A, Liberalesso PB, Vieira SC, Cordeiro ML. J Inherit Metab Dis; 2008 Dec 21; 31 Suppl 2():S357-61. PubMed ID: 18937049 [Abstract] [Full Text] [Related]
14. Effects of miglustat on stabilization of neurological disorder in niemann-pick disease type C: Iranian pediatric case series. Karimzadeh P, Tonekaboni SH, Ashrafi MR, Shafeghati Y, Rezayi A, Salehpour S, Ghofrani M, Taghdiri MM, Rahmanifar A, Zaman T, Aryani O, Shoar BN, Shiva F, Tavasoli A, Houshmand M. J Child Neurol; 2013 Dec 21; 28(12):1599-606. PubMed ID: 23143717 [Abstract] [Full Text] [Related]
15. Longitudinal changes in cerebellar and subcortical volumes in adult-onset Niemann-Pick disease type C patients treated with miglustat. Bowman EA, Walterfang M, Abel L, Desmond P, Fahey M, Velakoulis D. J Neurol; 2015 Sep 21; 262(9):2106-14. PubMed ID: 26092521 [Abstract] [Full Text] [Related]
19. Association of Miglustat With Swallowing Outcomes in Niemann-Pick Disease, Type C1. Solomon BI, Smith AC, Sinaii N, Farhat N, King MC, Machielse L, Porter FD. JAMA Neurol; 2020 Dec 01; 77(12):1564-1568. PubMed ID: 32897301 [Abstract] [Full Text] [Related]