Related Experiment Videos
[Transplant of hematopoietic stem cells in X-linked adrenoleukodystrophy]
1Sección Onco-hematología/trasplante hematopoyético; Hospital Niño Jesús, Madrid, 28009, España. lmadero@ctv.es
Revista De Neurologia
|October 6, 2001
Abstract:
Over 130 patients with adrenoleukodystrophy have received hematopoietic stem cell transplantation from normal donors. Without treatment, this disease has an inexorable fatal course. On the other hand, all of the engrafted hosts have had a remarkable positive clinical improvement in response to normalization of previously deficient enzymatic activity. The specific indications and methods for transplantation are also included in this article.