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4. Identification of seven novel SMPD1 mutations causing Niemann-Pick disease types A and B. Irun P; Mallén M; Dominguez C; Rodriguez-Sureda V; Alvarez-Sala LA; Arslan N; Bermejo N; Guerrero C; Perez de Soto I; Villalón L; Giraldo P; Pocovi M Clin Genet; 2013 Oct; 84(4):356-61. PubMed ID: 23252888 [TBL] [Abstract][Full Text] [Related]
5. Systemic lupus erythematosus occurring in a patient with Niemann-Pick type B disease. Murgia G; Firinu D; Meleddu R; Lorrai MM; Manconi PE; Del Giacco SR Lupus; 2015 Oct; 24(12):1332-4. PubMed ID: 25966928 [TBL] [Abstract][Full Text] [Related]
6. Limited benefits of presymptomatic cord blood transplantation in neurovisceral acid sphingomyelinase deficiency (ASMD) intermediate type. Mercati O; Pichard S; Ouachée M; Froissart R; Fenneteau O; Roche B; Elmaleh-Bergès M; Bertrand Y; Ogier de Baulny H; Vanier MT; Schiff M Eur J Paediatr Neurol; 2017 Nov; 21(6):907-911. PubMed ID: 28801223 [TBL] [Abstract][Full Text] [Related]
7. Spectrum of SMPD1 mutations in Asian-Indian patients with acid sphingomyelinase (ASM)-deficient Niemann-Pick disease. Ranganath P; Matta D; Bhavani GS; Wangnekar S; Jain JM; Verma IC; Kabra M; Puri RD; Danda S; Gupta N; Girisha KM; Sankar VH; Patil SJ; Ramadevi AR; Bhat M; Gowrishankar K; Mandal K; Aggarwal S; Tamhankar PM; Tilak P; Phadke SR; Dalal A Am J Med Genet A; 2016 Oct; 170(10):2719-30. PubMed ID: 27338287 [TBL] [Abstract][Full Text] [Related]
8. Niemann-Pick disease A or B in four pediatric patients and SMPD1 mutation carrier frequency in the Mexican population. Cerón-Rodríguez M; Vázquez-Martínez ER; García-Delgado C; Ortega-Vázquez A; Valencia-Mayoral P; Ramírez-Devars L; Arias-Villegas C; Monroy-Muñoz IE; López M; Cervantes A; Cerbón M; Morán-Barroso VF Ann Hepatol; 2019; 18(4):613-619. PubMed ID: 31122880 [TBL] [Abstract][Full Text] [Related]
9. Disease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD). McGovern MM; Avetisyan R; Sanson BJ; Lidove O Orphanet J Rare Dis; 2017 Feb; 12(1):41. PubMed ID: 28228103 [TBL] [Abstract][Full Text] [Related]
10. 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]
11. Analysis of the sphingomyelin phosphodiesterase 1 gene (SMPD1) in Turkish Niemann-Pick disease patients: mutation profile and description of a novel mutation. Aykut A; Karaca E; Onay H; Ucar SK; Coker M; Cogulu O; Ozkinay F Gene; 2013 Sep; 526(2):484-6. PubMed ID: 23618813 [TBL] [Abstract][Full Text] [Related]
12. Acid sphingomyelinase: identification of nine novel mutations among Italian Niemann Pick type B patients and characterization of in vivo functional in-frame start codon. Pittis MG; Ricci V; Guerci VI; Marçais C; Ciana G; Dardis A; Gerin F; Stroppiano M; Vanier MT; Filocamo M; Bembi B Hum Mutat; 2004 Aug; 24(2):186-7. PubMed ID: 15241805 [TBL] [Abstract][Full Text] [Related]
13. Identification of a distinct mutation spectrum in the SMPD1 gene of Chinese patients with acid sphingomyelinase-deficient Niemann-Pick disease. Zhang H; Wang Y; Gong Z; Li X; Qiu W; Han L; Ye J; Gu X Orphanet J Rare Dis; 2013 Jan; 8():15. PubMed ID: 23356216 [TBL] [Abstract][Full Text] [Related]
14. [Acid sphingomyelinase deficiency (Niemann-Pick disease type B) in adulthood: A retrospective multicentric study of 28 adult cases]. Lidove O; Belmatoug N; Froissart R; Lavigne C; Durieu I; Mazodier K; Serratrice C; Douillard C; Goizet C; Cathebras P; Besson G; Amoura Z; Tazi A; Gatfossé M; Rivière S; Sené T; Vanier MT; Ziza JM Rev Med Interne; 2017 May; 38(5):291-299. PubMed ID: 27884455 [TBL] [Abstract][Full Text] [Related]
15. Niemann-Pick disease type A and B are clinically but also enzymatically heterogeneous: pitfall in the laboratory diagnosis of sphingomyelinase deficiency associated with the mutation Q292 K. Harzer K; Rolfs A; Bauer P; Zschiesche M; Mengel E; Backes J; Kustermann-Kuhn B; Bruchelt G; van Diggelen OP; Mayrhofer H; Krägeloh-Mann I Neuropediatrics; 2003 Dec; 34(6):301-6. PubMed ID: 14681755 [TBL] [Abstract][Full Text] [Related]
16. Molecular analysis of the acid sphingomyelinase deficiency in a family with an intermediate form of Niemann-Pick disease. Ferlinz K; Hurwitz R; Weiler M; Suzuki K; Sandhoff K; Vanier MT Am J Hum Genet; 1995 Jun; 56(6):1343-9. PubMed ID: 7762557 [TBL] [Abstract][Full Text] [Related]
18. Reliable Assay of Acid Sphingomyelinase Deficiency with the Mutation Q292K by Tandem Mass Spectrometry. Ghomashchi F; Barcenas M; Turecek F; Scott CR; Gelb MH Clin Chem; 2015 May; 61(5):771-2. PubMed ID: 25770139 [No Abstract] [Full Text] [Related]
19. Niemann-Pick type B disease. Identification of a single codon deletion in the acid sphingomyelinase gene and genotype/phenotype correlations in type A and B patients. Levran O; Desnick RJ; Schuchman EH J Clin Invest; 1991 Sep; 88(3):806-10. PubMed ID: 1885770 [TBL] [Abstract][Full Text] [Related]
20. Preimplantation genetic diagnosis for Niemann-Pick disease type B. Hellani A; Schuchman EH; Al-Odaib A; Al Aqueel A; Jaroudi K; Ozand P; Coskun S Prenat Diagn; 2004 Dec; 24(12):943-8. PubMed ID: 15612058 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]