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

Neurogenetics Branch, National Institute of Neurological Disorders and Stroke/NIH, Bethesda, MD 20892, USA.
Molecular Genetics and Metabolism (Impact Factor: 2.83). 10/2010; 102(2):207-9. DOI: 10.1016/j.ymgme.2010.10.002
Source: PubMed

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.

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