Literature DB >> 7965430

Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation.

G Pridjian1, J Humbert, J Willis, E Shapira.   

Abstract

At 8 months of age, before clinical neurologic deterioration, the younger of two sisters with metachromatic leukodystrophy received a transplant of bone marrow from her haploidentical, heterozygote mother. Compared with the course in the older, affected, untreated sibling, the onset of neurologic regression was delayed 1 year and progressed at a slower rate.

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Year:  1994        PMID： 7965430     DOI： 10.1016/s0022-3476(94)70072-9

Source DB:  PubMed          Journal:  J Pediatr        ISSN： 0022-3476            Impact factor:   4.406


  5 in total

Review 1.  Gene transfer approaches to the lysosomal storage disorders.

Authors:  J A Barranger; E O Rice; W P Swaney
Journal:  Neurochem Res       Date:  1999-04       Impact factor: 3.996

Review 2.  The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome.

Authors:  W Krivit; L A Lockman; P A Watkins; J Hirsch; E G Shapiro
Journal:  J Inherit Metab Dis       Date:  1995       Impact factor: 4.982

Review 3.  Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation.

Authors:  E G Shapiro; L A Lockman; M Balthazor; W Krivit
Journal:  J Inherit Metab Dis       Date:  1995       Impact factor: 4.982

4.  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

Review 5.  Novel therapies for mucopolysaccharidosis type III.

Authors:  Berna Seker Yilmaz; James Davison; Simon A Jones; Julien Baruteau
Journal:  J Inherit Metab Dis       Date:  2020-09-28       Impact factor: 4.982
  5 in total

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