These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
281 related articles for article (PubMed ID: 37078237)
21. Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA). Chandler RJ; Venditti CP Mol Genet Metab; 2012 Nov; 107(3):617-9. PubMed ID: 23046887 [TBL] [Abstract][Full Text] [Related]
22. Systematic literature review and meta-analysis on the epidemiology of methylmalonic acidemia (MMA) with a focus on MMA caused by methylmalonyl-CoA mutase (mut) deficiency. Almási T; Guey LT; Lukacs C; Csetneki K; Vokó Z; Zelei T Orphanet J Rare Dis; 2019 Apr; 14(1):84. PubMed ID: 31023387 [TBL] [Abstract][Full Text] [Related]
23. Understanding acute metabolic decompensation in propionic and methylmalonic acidemias: a deep metabolic phenotyping approach. Haijes HA; Jans JJM; van der Ham M; van Hasselt PM; Verhoeven-Duif NM Orphanet J Rare Dis; 2020 Mar; 15(1):68. PubMed ID: 32143654 [TBL] [Abstract][Full Text] [Related]
24. Retrospective evaluation of the Dutch pre-newborn screening cohort for propionic acidemia and isolated methylmalonic acidemia: What to aim, expect, and evaluate from newborn screening? Haijes HA; Molema F; Langeveld M; Janssen MC; Bosch AM; van Spronsen F; Mulder MF; Verhoeven-Duif NM; Jans JJM; van der Ploeg AT; Wagenmakers MA; Rubio-Gozalbo ME; Brouwers MCGJ; de Vries MC; Langendonk JG; Williams M; van Hasselt PM J Inherit Metab Dis; 2020 May; 43(3):424-437. PubMed ID: 31828787 [TBL] [Abstract][Full Text] [Related]
26. Review of neuropsychological outcomes in isolated methylmalonic acidemia: recommendations for assessing impact of treatments. Waisbren SE Metab Brain Dis; 2022 Jun; 37(5):1317-1335. PubMed ID: 35348993 [TBL] [Abstract][Full Text] [Related]
27. Liver transplantation in propionic and methylmalonic acidemia: A single center study with literature review. Pillai NR; Stroup BM; Poliner A; Rossetti L; Rawls B; Shayota BJ; Soler-Alfonso C; Tunuguntala HP; Goss J; Craigen W; Scaglia F; Sutton VR; Himes RW; Burrage LC Mol Genet Metab; 2019 Dec; 128(4):431-443. PubMed ID: 31757659 [TBL] [Abstract][Full Text] [Related]
28. [Methylmalonic acidemia. Diagnosis and treatment in various patients]. Bakker HD; Duran M; van Gennip AH; van Sprang FJ; Wadman SK Ned Tijdschr Geneeskd; 1978 May; 122(20):707-12. PubMed ID: 26029 [No Abstract] [Full Text] [Related]
29. [Mutation analysis of the methylmalonyl-CoA mutase gene in ten Mexican patients with methylmalonic acidemia]. Méndez ST; Vela-Amieva M; Velázquez-Arellano A; Ibarra I; Flores ME Rev Invest Clin; 2012; 64(3):255-61. PubMed ID: 23045948 [TBL] [Abstract][Full Text] [Related]
30. Propionic and methylmalonic acidemia: antisense therapeutics for intronic variations causing aberrantly spliced messenger RNA. Rincón A; Aguado C; Desviat LR; Sánchez-Alcudia R; Ugarte M; Pérez B Am J Hum Genet; 2007 Dec; 81(6):1262-70. PubMed ID: 17966092 [TBL] [Abstract][Full Text] [Related]
31. The molecular landscape of propionic acidemia and methylmalonic aciduria in Latin America. Pérez B; Angaroni C; Sánchez-Alcudia R; Merinero B; Pérez-Cerdá C; Specola N; Rodríguez-Pombo P; Wajner M; de Kremer RD; Cornejo V; Desviat LR; Ugarte M J Inherit Metab Dis; 2010 Oct; 33(Suppl 2):S307-14. PubMed ID: 20549364 [TBL] [Abstract][Full Text] [Related]
32. Identification of 2,2-Dimethylbutanoic Acid (HST5040), a Clinical Development Candidate for the Treatment of Propionic Acidemia and Methylmalonic Acidemia. Armstrong AJ; Henke BR; Collado MS; Taylor JM; Pourtaheri TD; Dillberger JE; Roper TD; Wamhoff BR; Olson MW; Figler RA; Hoang SA; Reardon JE; Johns BA J Med Chem; 2021 Apr; 64(8):5037-5048. PubMed ID: 33848153 [TBL] [Abstract][Full Text] [Related]
33. Long-term effectiveness of carglumic acid in patients with propionic acidemia (PA) and methylmalonic acidemia (MMA): a randomized clinical trial. Alfadhel M; Nashabat M; Saleh M; Elamin M; Alfares A; Al Othaim A; Umair M; Ahmed H; Ababneh F; Al Mutairi F; Eyaid W; Alswaid A; Alohali L; Faqeih E; Almannai M; Aljeraisy M; Albdah B; Hussein MA; Rahbeeni Z; Alasmari A Orphanet J Rare Dis; 2021 Oct; 16(1):422. PubMed ID: 34635114 [TBL] [Abstract][Full Text] [Related]
34. Methylmalonic acidemia/propionic acidemia - the biochemical presentation and comparing the outcome between liver transplantation versus non-liver transplantation groups. Chu TH; Chien YH; Lin HY; Liao HC; Ho HJ; Lai CJ; Chiang CC; Lin NC; Yang CF; Hwu WL; Lee NC; Lin SP; Liu CS; Hu RH; Ho MC; Niu DM Orphanet J Rare Dis; 2019 Apr; 14(1):73. PubMed ID: 30940196 [TBL] [Abstract][Full Text] [Related]
35. Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes. Chandler RJ; Tsai MS; Dorko K; Sloan J; Korson M; Freeman R; Strom S; Venditti CP BMC Med Genet; 2007 Apr; 8():24. PubMed ID: 17470278 [TBL] [Abstract][Full Text] [Related]
36. Plasma methylcitric acid and its correlations with other disease biomarkers: The impact in the follow up of patients with propionic and methylmalonic acidemia. Maines E; Catesini G; Boenzi S; Mosca A; Candusso M; Dello Strologo L; Martinelli D; Maiorana A; Liguori A; Olivieri G; Taurisano R; Piemonte F; Rizzo C; Spada M; Dionisi-Vici C J Inherit Metab Dis; 2020 Nov; 43(6):1173-1185. PubMed ID: 32681732 [TBL] [Abstract][Full Text] [Related]
37. Long-term liver disease in methylmalonic and propionic acidemias. Imbard A; Garcia Segarra N; Tardieu M; Broué P; Bouchereau J; Pichard S; de Baulny HO; Slama A; Mussini C; Touati G; Danjoux M; Gaignard P; Vogel H; Labarthe F; Schiff M; Benoist JF Mol Genet Metab; 2018 Apr; 123(4):433-440. PubMed ID: 29433791 [TBL] [Abstract][Full Text] [Related]
38. Improved therapeutic efficacy in two mouse models of methylmalonic acidemia (MMA) using a second-generation mRNA therapy. Coughlan KA; Eybye M; Henderson N; DeAntonis CM; Frassetto A; Hanahoe E; Ketova T; Jacquinet E; Presnyak V; Jain R; Marshall J; Martini PGV Mol Genet Metab; 2024; 143(1-2):108560. PubMed ID: 39121792 [TBL] [Abstract][Full Text] [Related]
39. Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia. Chandler RJ; Venturoni LE; Liao J; Hubbard BT; Schneller JL; Hoffmann V; Gordo S; Zang S; Ko CW; Chau N; Chiang K; Kay MA; Barzel A; Venditti CP Hepatology; 2021 Jun; 73(6):2223-2237. PubMed ID: 32976669 [TBL] [Abstract][Full Text] [Related]
40. Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemia. Manoli I; Sysol JR; Li L; Houillier P; Garone C; Wang C; Zerfas PM; Cusmano-Ozog K; Young S; Trivedi NS; Cheng J; Sloan JL; Chandler RJ; Abu-Asab M; Tsokos M; Elkahloun AG; Rosen S; Enns GM; Berry GT; Hoffmann V; DiMauro S; Schnermann J; Venditti CP Proc Natl Acad Sci U S A; 2013 Aug; 110(33):13552-7. PubMed ID: 23898205 [TBL] [Abstract][Full Text] [Related] [Previous] [Next] [New Search]