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Items: 1 to 20 of 21

1.

Metachromatic leukodystrophy: a scoring system for brain MR imaging observations.

Eichler F, Grodd W, Grant E, Sessa M, Biffi A, Bley A, Kohlschuetter A, Loes DJ, Kraegeloh-Mann I.

AJNR Am J Neuroradiol. 2009 Nov;30(10):1893-7. doi: 10.3174/ajnr.A1739. Epub 2009 Oct 1.

2.

Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy.

Pierson TM, Bonnemann CG, Finkel RS, Bunin N, Tennekoon GI.

Ann Neurol. 2008 Nov;64(5):583-7. doi: 10.1002/ana.21522.

3.

Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options.

Gieselmann V.

Acta Paediatr. 2008 Apr;97(457):15-21. doi: 10.1111/j.1651-2227.2008.00648.x.

PMID:
18339182
4.

Stabilization of juvenile metachromatic leukodystrophy after bone marrow transplantation: a 13-year follow-up.

Görg M, Wilck W, Granitzny B, Suerken A, Lukacs Z, Ding X, Schulte-Markwort M, Kohlschütter A.

J Child Neurol. 2007 Sep;22(9):1139-42.

PMID:
17890417
5.

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.

Biffi A, Capotondo A, Fasano S, del Carro U, Marchesini S, Azuma H, Malaguti MC, Amadio S, Brambilla R, Grompe M, Bordignon C, Quattrini A, Naldini L.

J Clin Invest. 2006 Nov;116(11):3070-82.

6.

Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases.

Martin PL, Carter SL, Kernan NA, Sahdev I, Wall D, Pietryga D, Wagner JE, Kurtzberg J.

Biol Blood Marrow Transplant. 2006 Feb;12(2):184-94.

7.

Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease.

Escolar ML, Poe MD, Provenzale JM, Richards KC, Allison J, Wood S, Wenger DA, Pietryga D, Wall D, Champagne M, Morse R, Krivit W, Kurtzberg J.

N Engl J Med. 2005 May 19;352(20):2069-81.

8.

Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy.

Matzner U, Herbst E, Hedayati KK, Lüllmann-Rauch R, Wessig C, Schröder S, Eistrup C, Möller C, Fogh J, Gieselmann V.

Hum Mol Genet. 2005 May 1;14(9):1139-52. Epub 2005 Mar 16.

PMID:
15772092
9.

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines.

Peters C, Steward CG; National Marrow Donor Program; International Bone Marrow Transplant Registry; Working Party on Inborn Errors, European Bone Marrow Transplant Group.

Bone Marrow Transplant. 2003 Feb;31(4):229-39. Review.

PMID:
12621457
10.

The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease.

Altarescu G, Hill S, Wiggs E, Jeffries N, Kreps C, Parker CC, Brady RO, Barton NW, Schiffmann R.

J Pediatr. 2001 Apr;138(4):539-47.

PMID:
11295718
11.

Improved peripheral nerve conduction, EEG and verbal IQ after bone marrow transplantation for adult metachromatic leukodystrophy.

Solders G, Celsing G, Hagenfeldt L, Ljungman P, Isberg B, Ringdén O.

Bone Marrow Transplant. 1998 Dec;22(11):1119-22.

12.

Long-term stabilization after bone marrow transplantation in juvenile metachromatic leukodystrophy.

Kidd D, Nelson J, Jones F, Dusoir H, Wallace I, McKinstry S, Patterson V.

Arch Neurol. 1998 Jan;55(1):98-9. No abstract available.

PMID:
9443716
13.

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.

PMID:
8807106
14.

Late juvenile metachromatic leukodystrophy treated with bone marrow transplantation; a 4-year follow-up study.

Navarro C, Fernández JM, Domínguez C, Fachal C, Alvarez M.

Neurology. 1996 Jan;46(1):254-6.

PMID:
8559389
15.

Electrophysiologic correlates of peripheral nervous system maturation in infancy and childhood.

Parano E, Uncini A, De Vivo DC, Lovelace RE.

J Child Neurol. 1993 Oct;8(4):336-8.

PMID:
8228028
16.

Molecular genetics of metachromatic leukodystrophy.

Gieselmann V, Zlotogora J, Harris A, Wenger DA, Morris CP.

Hum Mutat. 1994;4(4):233-42. Review.

PMID:
7866401
17.
18.

Juvenile metachromatic leukodystrophy: neurological outcome two years after bone marrow transplantation.

Guffon N, Souillet G, Maire I, Dorche C, Mathieu M, Guibaud P.

J Inherit Metab Dis. 1995;18(2):159-61. No abstract available.

PMID:
7564235
19.

The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome.

Krivit W, Lockman LA, Watkins PA, Hirsch J, Shapiro EG.

J Inherit Metab Dis. 1995;18(4):398-412. Review.

PMID:
7494399
20.

Possible pathways for lysosomal enzyme delivery.

Geuze HJ, Slot JW, Strous GJ, Hasilik A, von Figura K.

J Cell Biol. 1985 Dec;101(6):2253-62.

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