BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

445 related articles for article (PubMed ID: 25987178)

  • 1. Metachromatic leukodystrophy: Disease spectrum and approaches for treatment.
    van Rappard DF; Boelens JJ; Wolf NI
    Best Pract Res Clin Endocrinol Metab; 2015 Mar; 29(2):261-73. PubMed ID: 25987178
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.
    Sessa M; Lorioli L; Fumagalli F; Acquati S; Redaelli D; Baldoli C; Canale S; Lopez ID; Morena F; Calabria A; Fiori R; Silvani P; Rancoita PM; Gabaldo M; Benedicenti F; Antonioli G; Assanelli A; Cicalese MP; Del Carro U; Sora MG; Martino S; Quattrini A; Montini E; Di Serio C; Ciceri F; Roncarolo MG; Aiuti A; Naldini L; Biffi A
    Lancet; 2016 Jul; 388(10043):476-87. PubMed ID: 27289174
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Peripheral neuropathy in metachromatic leukodystrophy: current status and future perspective.
    Beerepoot S; Nierkens S; Boelens JJ; Lindemans C; Bugiani M; Wolf NI
    Orphanet J Rare Dis; 2019 Nov; 14(1):240. PubMed ID: 31684987
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Developing treatment options for metachromatic leukodystrophy.
    Batzios SP; Zafeiriou DI
    Mol Genet Metab; 2012 Jan; 105(1):56-63. PubMed ID: 22078456
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Gene therapy in metachromatic leukodystrophy.
    Sevin C; Cartier-Lacave N; Aubourg P
    Int J Clin Pharmacol Ther; 2009; 47 Suppl 1():S128-31. PubMed ID: 20040324
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Intestinal involvement in metachromatic leukodystrophy.
    Yavuz H; Yüksekkaya HA
    J Child Neurol; 2011 Jan; 26(1):117-20. PubMed ID: 21212458
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
    Biffi A; Montini E; Lorioli L; Cesani M; Fumagalli F; Plati T; Baldoli C; Martino S; Calabria A; Canale S; Benedicenti F; Vallanti G; Biasco L; Leo S; Kabbara N; Zanetti G; Rizzo WB; Mehta NA; Cicalese MP; Casiraghi M; Boelens JJ; Del Carro U; Dow DJ; Schmidt M; Assanelli A; Neduva V; Di Serio C; Stupka E; Gardner J; von Kalle C; Bordignon C; Ciceri F; Rovelli A; Roncarolo MG; Aiuti A; Sessa M; Naldini L
    Science; 2013 Aug; 341(6148):1233158. PubMed ID: 23845948
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Developing therapeutic approaches for metachromatic leukodystrophy.
    Patil SA; Maegawa GH
    Drug Des Devel Ther; 2013; 7():729-45. PubMed ID: 23966770
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Lysosomal leukodystrophies: Krabbe disease and metachromatic leukodystrophy.
    Kohlschütter A
    Handb Clin Neurol; 2013; 113():1611-8. PubMed ID: 23622382
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Enzyme, cell and gene-based therapies for metachromatic leukodystrophy.
    Sevin C; Aubourg P; Cartier N
    J Inherit Metab Dis; 2007 Apr; 30(2):175-83. PubMed ID: 17347913
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Atidarsagene autotemcel for metachromatic leukodystrophy.
    Messina M; Gissen P
    Drugs Today (Barc); 2023 Feb; 59(2):63-70. PubMed ID: 36811406
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Metachromatic leukodystrophy--an update.
    Gieselmann V; Krägeloh-Mann I
    Neuropediatrics; 2010 Feb; 41(1):1-6. PubMed ID: 20571983
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Neurodegenerative disease after hematopoietic stem cell transplantation in metachromatic leukodystrophy.
    Al-Saady M; Beerepoot S; Plug BC; Breur M; Galabova H; Pouwels PJW; Boelens JJ; Lindemans C; van Hasselt PM; Matzner U; Vanderver A; Bugiani M; van der Knaap MS; Wolf NI
    Ann Clin Transl Neurol; 2023 Jul; 10(7):1146-1159. PubMed ID: 37212343
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Evolutionary redesign of the lysosomal enzyme arylsulfatase A increases efficacy of enzyme replacement therapy for metachromatic leukodystrophy.
    Simonis H; Yaghootfam C; Sylvester M; Gieselmann V; Matzner U
    Hum Mol Genet; 2019 Jun; 28(11):1810-1821. PubMed ID: 30657900
    [TBL] [Abstract][Full Text] [Related]  

  • 15. An unusual homozygous arylsulfatase: a pseudodeficiency in a metachromatic leukodystrophy Tunisian patient.
    Tinsa F; Caillaud C; Vanier MT; Bousnina D; Boussetta K; Bousnina S
    J Child Neurol; 2010 Jan; 25(1):82-6. PubMed ID: 19574581
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Peripheral neuropathy as the sole initial finding in three children with infantile metachromatic leukodystrophy.
    Haberlandt E; Scholl-Bürgi S; Neuberger J; Felber S; Gotwald T; Sauter R; Rostasy K; Karall D; Korinthenberg R
    Eur J Paediatr Neurol; 2009 May; 13(3):257-60. PubMed ID: 18571943
    [TBL] [Abstract][Full Text] [Related]  

  • 17. [Metachromatic Leukodystrophy (MLD): MRI findings].
    Fenchel M; Hauser TK; Nägele T; Ernemann U; Horger M
    Rofo; 2011 Oct; 183(10):893-5. PubMed ID: 22232791
    [No Abstract]   [Full Text] [Related]  

  • 18. Brain cell type-specific endocytosis of arylsulfatase A identifies limitations of enzyme-based therapies for metachromatic leukodystrophy.
    Kaminski D; Yaghootfam C; Matthes F; Reßing A; Gieselmann V; Matzner U
    Hum Mol Genet; 2021 Feb; 29(23):3807-3817. PubMed ID: 33367737
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options.
    Gieselmann V
    Acta Paediatr; 2008 Apr; 97(457):15-21. PubMed ID: 18339182
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Hematopoietic SCT: a useful treatment for late metachromatic leukodystrophy.
    Solders M; Martin DA; Andersson C; Remberger M; Andersson T; Ringdén O; Solders G
    Bone Marrow Transplant; 2014 Aug; 49(8):1046-51. PubMed ID: 24797185
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 23.