TITLE

Early Use of Drastic Therapy

AUTHOR(S)
Weinberg, Kenneth I.
PUB. DATE
May 2005
SOURCE
New England Journal of Medicine;5/19/2005, Vol. 352 Issue 20, p2124
SOURCE TYPE
Academic Journal
DOC. TYPE
Editorial
ABSTRACT
Focuses on the treatment of Krabbe's disease, also known as globoid-cell leukodystrophy. Claim that Krabbe's disease is an inborn error of lipid metabolism; Impact of Krabbe's disease on the central nervous system; Potential for improved status following enzyme replacement; Details of the treatment of Krabbe's disease with hematopoietic stem-cell transplantation; Role of hematopoietic stem-cell transplantation in the treatment of diseases of the central nervous system; Reference to a study on the treatment of Krabbe's disease in infants; Observation of improved survival and symptoms in infants treated with hematopoietic stem-cell transplantation; Implications of the study for the treatment of pediatric disease.
ACCESSION #
24926049

 

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