BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

202 related articles for article (PubMed ID: 24499656)

  • 1. Language and cognition in children with metachromatic leukodystrophy: onset and natural course in a nationwide cohort.
    Kehrer C; Groeschel S; Kustermann-Kuhn B; Bürger F; Köhler W; Kohlschütter A; Bley A; Steinfeld R; Gieselmann V; Krägeloh-Mann I;
    Orphanet J Rare Dis; 2014 Feb; 9():18. PubMed ID: 24499656
    [TBL] [Abstract][Full Text] [Related]  

  • 2. The natural course of gross motor deterioration in metachromatic leukodystrophy.
    Kehrer C; Blumenstock G; Gieselmann V; Krägeloh-Mann I;
    Dev Med Child Neurol; 2011 Sep; 53(9):850-855. PubMed ID: 21707604
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Insights into the natural history of metachromatic leukodystrophy from interviews with caregivers.
    Harrington M; Whalley D; Twiss J; Rushton R; Martin S; Huynh L; Yang H
    Orphanet J Rare Dis; 2019 Apr; 14(1):89. PubMed ID: 31036045
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Metachromatic leukodystrophy: natural course of cerebral MRI changes in relation to clinical course.
    Groeschel S; Kehrer C; Engel C; I Dali C; Bley A; Steinfeld R; Grodd W; Krägeloh-Mann I
    J Inherit Metab Dis; 2011 Oct; 34(5):1095-102. PubMed ID: 21698385
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Juvenile Metachromatic Leukodystrophy Compared With Nontransplanted Control Patients.
    Groeschel S; Kühl JS; Bley AE; Kehrer C; Weschke B; Döring M; Böhringer J; Schrum J; Santer R; Kohlschütter A; Krägeloh-Mann I; Müller I
    JAMA Neurol; 2016 Sep; 73(9):1133-40. PubMed ID: 27400410
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy.
    Martin HR; Poe MD; Provenzale JM; Kurtzberg J; Mendizabal A; Escolar ML
    Biol Blood Marrow Transplant; 2013 Apr; 19(4):616-24. PubMed ID: 23348427
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Phenotypic variation between siblings with Metachromatic Leukodystrophy.
    Elgün S; Waibel J; Kehrer C; van Rappard D; Böhringer J; Beck-Wödl S; Just J; Schöls L; Wolf N; Krägeloh-Mann I; Groeschel S
    Orphanet J Rare Dis; 2019 Jun; 14(1):136. PubMed ID: 31186049
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.
    Fumagalli F; Zambon AA; Rancoita PMV; Baldoli C; Canale S; Spiga I; Medaglini S; Penati R; Facchini M; Ciotti F; Sarzana M; Lorioli L; Cesani M; Natali Sora MG; Del Carro U; Cugnata F; Antonioli G; Recupero S; Calbi V; Di Serio C; Aiuti A; Biffi A; Sessa M
    J Inherit Metab Dis; 2021 Sep; 44(5):1151-1164. PubMed ID: 33855715
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Understanding caregiver descriptions of initial signs and symptoms to improve diagnosis of metachromatic leukodystrophy.
    Eichler F; Sevin C; Barth M; Pang F; Howie K; Walz M; Wilds A; Calcagni C; Chanson C; Campbell L
    Orphanet J Rare Dis; 2022 Oct; 17(1):370. PubMed ID: 36195888
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report.
    Boucher AA; Miller W; Shanley R; Ziegler R; Lund T; Raymond G; Orchard PJ
    Orphanet J Rare Dis; 2015 Aug; 10():94. PubMed ID: 26245762
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Clinical outcome in four children with metachromatic leukodystrophy treated by bone marrow transplantation.
    Malm G; Ringdén O; Winiarski J; Gröndahl E; Uyebrant P; Eriksson U; Håkansson H; Skjeldal O; Månsson JE
    Bone Marrow Transplant; 1996 Jun; 17(6):1003-8. PubMed ID: 8807106
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
    Fumagalli F; Calbi V; Natali Sora MG; Sessa M; Baldoli C; Rancoita PMV; Ciotti F; Sarzana M; Fraschini M; Zambon AA; Acquati S; Redaelli D; Attanasio V; Miglietta S; De Mattia F; Barzaghi F; Ferrua F; Migliavacca M; Tucci F; Gallo V; Del Carro U; Canale S; Spiga I; Lorioli L; Recupero S; Fratini ES; Morena F; Silvani P; Calvi MR; Facchini M; Locatelli S; Corti A; Zancan S; Antonioli G; Farinelli G; Gabaldo M; Garcia-Segovia J; Schwab LC; Downey GF; Filippi M; Cicalese MP; Martino S; Di Serio C; Ciceri F; Bernardo ME; Naldini L; Biffi A; Aiuti A
    Lancet; 2022 Jan; 399(10322):372-383. PubMed ID: 35065785
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Association of Age at Onset and First Symptoms With Disease Progression in Patients With Metachromatic Leukodystrophy.
    Kehrer C; Elgün S; Raabe C; Böhringer J; Beck-Wödl S; Bevot A; Kaiser N; Schöls L; Krägeloh-Mann I; Groeschel S
    Neurology; 2021 Jan; 96(2):e255-e266. PubMed ID: 33046606
    [TBL] [Abstract][Full Text] [Related]  

  • 14. The natural history and burden of illness of metachromatic leukodystrophy: a systematic literature review.
    Chang SC; Eichinger CS; Field P
    Eur J Med Res; 2024 Mar; 29(1):181. PubMed ID: 38494502
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Evaluating meaningful changes in physical functioning and cognitive declines in metachromatic leukodystrophy: a caregiver interview study.
    Martin S; Harris N; Romanus D
    J Patient Rep Outcomes; 2023 Jul; 7(1):70. PubMed ID: 37458805
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Metachromatic leucodystrophy: review of 38 cases.
    MacFaul R; Cavanagh N; Lake BD; Stephens R; Whitfield AE
    Arch Dis Child; 1982 Mar; 57(3):168-75. PubMed ID: 7073297
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Clinical and biochemical study of 29 Brazilian patients with metachromatic leukodystrophy.
    Artigalás O; Lagranha VL; Saraiva-Pereira ML; Burin MG; Lourenço CM; van der Linden H; Santos ML; Rosemberg S; Steiner CE; Kok F; de Souza CF; Jardim LB; Giugliani R; Schwartz IV
    J Inherit Metab Dis; 2010 Dec; 33 Suppl 3():S257-62. PubMed ID: 20596894
    [TBL] [Abstract][Full Text] [Related]  

  • 18. The importance of early diagnosis and views on newborn screening in metachromatic leukodystrophy: results of a Caregiver Survey in the UK and Republic of Ireland.
    Morton G; Thomas S; Roberts P; Clark V; Imrie J; Morrison A
    Orphanet J Rare Dis; 2022 Nov; 17(1):403. PubMed ID: 36329444
    [TBL] [Abstract][Full Text] [Related]  

  • 19. 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]  

  • 20.
    ; ; . PubMed ID:
    [No Abstract]   [Full Text] [Related]  

    [Next]    [New Search]
    of 11.