Literature DB >> 21035368

Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: a 5-year follow-up in three affected siblings.

Casey Cable1, Richard S Finkel, Tanya J Lehky, Nadia M Biassou, Edythe A Wiggs, Nancy Bunin, Tyler Mark Pierson.   

Abstract

Unrelated umbilical cord blood transplantation (UCBT) was used to treat three siblings with juvenile metachromatic leukodystrophy (jMLD). The efficacy of this therapy was measured over a 5-year period with serial neurological examinations, neuroimaging, nerve conduction studies (NCS), and neuropsychological evaluations (NPE). Outcomes were a function of disease stage at time of UCBT with alteration of disease course occurring in the first 2 years after UCBT and then subsequent halting of progression and stabilization of symptoms and disease. Published by Elsevier Inc.

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Year:  2010        PMID： 21035368      PMCID： PMC3053057          DOI： 10.1016/j.ymgme.2010.10.002

Source DB:  PubMed          Journal:  Mol Genet Metab        ISSN： 1096-7192            Impact factor:   4.797


  21 in total

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

Authors:  G Altarescu; S Hill; E Wiggs; N Jeffries; C Kreps; C C Parker; R O Brady; N W Barton; R Schiffmann
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Review 2.  The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome.

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Journal:  J Inherit Metab Dis       Date:  1995       Impact factor: 4.982

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

Authors:  N Guffon; G Souillet; I Maire; C Dorche; M Mathieu; P Guibaud
Journal:  J Inherit Metab Dis       Date:  1995       Impact factor: 4.982

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

Authors:  E Parano; A Uncini; D C De Vivo; R E Lovelace
Journal:  J Child Neurol       Date:  1993-10       Impact factor: 1.987

5.  Bone-marrow transplantation for metachromatic leucodystrophy.

Authors:  E Bayever; S Ladisch; M Philippart; N Brill; M Nuwer; R S Sparkes; S A Feig
Journal:  Lancet       Date:  1985-08-31       Impact factor: 79.321

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

Authors:  C Navarro; J M Fernández; C Domínguez; C Fachal; M Alvarez
Journal:  Neurology       Date:  1996-01       Impact factor: 9.910

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

Authors:  F Eichler; W Grodd; E Grant; M Sessa; A Biffi; A Bley; A Kohlschuetter; D J Loes; I Kraegeloh-Mann
Journal:  AJNR Am J Neuroradiol       Date:  2009-10-01       Impact factor: 3.825

8.  Sensory nerve conduction studies in children. Age-related changes of conduction velocities.

Authors:  O K Rabben
Journal:  Neuropediatrics       Date:  1995-02       Impact factor: 1.947

Review 9.  Molecular genetics of metachromatic leukodystrophy.

Authors:  V Gieselmann; J Zlotogora; A Harris; D A Wenger; C P Morris
Journal:  Hum Mutat       Date:  1994       Impact factor: 4.878

10.  Possible pathways for lysosomal enzyme delivery.

Authors:  H J Geuze; J W Slot; G J Strous; A Hasilik; K von Figura
Journal:  J Cell Biol       Date:  1985-12       Impact factor: 10.539
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  7 in total

Review 1.  Gene therapy for metachromatic leukodystrophy.

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2.  Disease specific therapies in leukodystrophies and leukoencephalopathies.

Authors:  Guy Helman; Keith Van Haren; Joshua L Bonkowsky; Genevieve Bernard; Amy Pizzino; Nancy Braverman; Dean Suhr; Marc C Patterson; S Ali Fatemi; Jeff Leonard; Marjo S van der Knaap; Stephen A Back; Stephen Damiani; Steven A Goldman; Asako Takanohashi; Magdalena Petryniak; David Rowitch; Albee Messing; Lawrence Wrabetz; Raphael Schiffmann; Florian Eichler; Maria L Escolar; Adeline Vanderver
Journal:  Mol Genet Metab       Date:  2015-02-07       Impact factor: 4.797

3.  Hematopoietic SCT: a useful treatment for late metachromatic leukodystrophy.

Authors:  M Solders; D A Martin; C Andersson; M Remberger; T Andersson; O Ringdén; G Solders
Journal:  Bone Marrow Transplant       Date:  2014-05-05       Impact factor: 5.483

Review 4.  Emerging treatments for pediatric leukodystrophies.

Authors:  Guy Helman; Keith Van Haren; Maria L Escolar; Adeline Vanderver
Journal:  Pediatr Clin North Am       Date:  2015-04-08       Impact factor: 3.278

5.  Phenotypic variation between siblings with Metachromatic Leukodystrophy.

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Review 6.  Peripheral neuropathy in metachromatic leukodystrophy: current status and future perspective.

Authors:  Shanice Beerepoot; Stefan Nierkens; Jaap Jan Boelens; Caroline Lindemans; Marianna Bugiani; Nicole I Wolf
Journal:  Orphanet J Rare Dis       Date:  2019-11-04       Impact factor: 4.123

7.  Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report.

Authors:  Alexander A Boucher; Weston Miller; Ryan Shanley; Richard Ziegler; Troy Lund; Gerald Raymond; Paul J Orchard
Journal:  Orphanet J Rare Dis       Date:  2015-08-07       Impact factor: 4.123
  7 in total

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