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4. Alterations of myelin-specific proteins and sphingolipids characterize the brains of acid sphingomyelinase-deficient mice, an animal model of Niemann-Pick disease type A. Buccinnà B; Piccinini M; Prinetti A; Scandroglio F; Prioni S; Valsecchi M; Votta B; Grifoni S; Lupino E; Ramondetti C; Schuchman EH; Giordana MT; Sonnino S; Rinaudo MT J Neurochem; 2009 Apr; 109(1):105-15. PubMed ID: 19187445 [TBL] [Abstract][Full Text] [Related]
5. Neurons regulate the esterification of bioactive lipid mediators in the brain of acid sphingomyelinase deficient mice. Taha AY; Gaudioso Á; Moran-Garrido M; Camunas-Alberca SM; Bachiller-Hernández J; Sáiz J; Ledesma MD; Barbas C Prog Neuropsychopharmacol Biol Psychiatry; 2024 Feb; 129():110896. PubMed ID: 37956788 [TBL] [Abstract][Full Text] [Related]
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8. Consensus clinical management guidelines for acid sphingomyelinase deficiency (Niemann-Pick disease types A, B and A/B). Geberhiwot T; Wasserstein M; Wanninayake S; Bolton SC; Dardis A; Lehman A; Lidove O; Dawson C; Giugliani R; Imrie J; Hopkin J; Green J; de Vicente Corbeira D; Madathil S; Mengel E; Ezgü F; Pettazzoni M; Sjouke B; Hollak C; Vanier MT; McGovern M; Schuchman E Orphanet J Rare Dis; 2023 Apr; 18(1):85. PubMed ID: 37069638 [TBL] [Abstract][Full Text] [Related]
9. Quantification of lysosphingomyelin and lysosphingomyelin-509 for the screening of acid sphingomyelinase deficiency. Kubaski F; Burlina A; Pereira D; Silva C; Herbst ZM; Trapp FB; Michelin-Tirelli K; Lopes FF; Burin MG; Brusius-Facchin AC; Netto ABO; Poletto E; Bernardes TM; Carvalho GS; Sorte NB; Ferreira FN; Perin N; Clivati MR; de Santana MTS; Lobos SFG; Leão EKEA; Coutinho MP; Pinos PV; Santos MLSF; Penatti DA; Lourenço CM; Polo G; Giugliani R Orphanet J Rare Dis; 2022 Nov; 17(1):407. PubMed ID: 36348386 [TBL] [Abstract][Full Text] [Related]
10. Massive Accumulation of Sphingomyelin Affects the Lysosomal and Mitochondria Compartments and Promotes Apoptosis in Niemann-Pick Disease Type A. Carsana EV; Lunghi G; Prioni S; Mauri L; Loberto N; Prinetti A; Zucca FA; Bassi R; Sonnino S; Chiricozzi E; Duga S; Straniero L; Asselta R; Soldà G; Samarani M; Aureli M J Mol Neurosci; 2022 Jul; 72(7):1482-1499. PubMed ID: 35727525 [TBL] [Abstract][Full Text] [Related]
11. Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy. Jones SA; McGovern M; Lidove O; Giugliani R; Mistry PK; Dionisi-Vici C; Munoz-Rojas MV; Nalysnyk L; Schecter AD; Wasserstein M Mol Genet Metab; 2020; 131(1-2):116-123. PubMed ID: 32616389 [TBL] [Abstract][Full Text] [Related]
12. Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD). Wasserstein M; Dionisi-Vici C; Giugliani R; Hwu WL; Lidove O; Lukacs Z; Mengel E; Mistry PK; Schuchman EH; McGovern M Mol Genet Metab; 2019 Feb; 126(2):98-105. PubMed ID: 30514648 [TBL] [Abstract][Full Text] [Related]
13. Importance to include differential diagnostics for acid sphingomyelinase deficiency (ASMD) in patients suspected to have to Gaucher disease. Oliva P; Schwarz M; Mechtler TP; Sansen S; Keutzer J; Prusa AR; Streubel B; Kasper DC Mol Genet Metab; 2023 May; 139(1):107563. PubMed ID: 37086570 [TBL] [Abstract][Full Text] [Related]
14. Similarities and differences between Gaucher disease and acid sphingomyelinase deficiency: An algorithm to support the diagnosis. Cappellini MD; Motta I; Barbato A; Giuffrida G; Manna R; Carubbi F; Giona F Eur J Intern Med; 2023 Feb; 108():81-84. PubMed ID: 36443133 [TBL] [Abstract][Full Text] [Related]
15. Neuropathology of the acid sphingomyelinase knockout mouse model of Niemann-Pick A disease including structure-function studies associated with cerebellar Purkinje cell degeneration. Macauley SL; Sidman RL; Schuchman EH; Taksir T; Stewart GR Exp Neurol; 2008 Dec; 214(2):181-92. PubMed ID: 18778708 [TBL] [Abstract][Full Text] [Related]
16. Acid Sphingomyelinase Deficiency Type B Patient-Derived Liver Organoids Reveals Altered Lysosomal Gene Expression and Lipid Homeostasis. Gomez-Mariano G; Perez-Luz S; Ramos-Del Saz S; Matamala N; Hernandez-SanMiguel E; Fernandez-Prieto M; Gil-Martin S; Justo I; Marcacuzco A; Martinez-Delgado B Int J Mol Sci; 2023 Aug; 24(16):. PubMed ID: 37628828 [TBL] [Abstract][Full Text] [Related]
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19. Novel mutations in the SMPD1 gene in Jordanian children with Acid sphingomyelinase deficiency (Niemann-Pick types A and B). Al-Eitan L; Alqa'qa' K; Amayreh W; Aljamal H; Khasawneh R; Al-Zoubi B; Okour I; Haddad A; Haddad Y; Haddad H Gene; 2020 Jul; 747():144683. PubMed ID: 32311413 [TBL] [Abstract][Full Text] [Related]