Inherited Metabolic Disease
Data on inherited metabolic disease reported by researchers at Institute of Metabolic Disease
Published in Gene Therapy Weekly, August 5th, 2010
A report, 'Therapeutic approaches for neuronopathic lysosomal storage disorders,' is newly published data in Journal of Inherited Metabolic Disease. "Therapy of the central nervous system (CNS) manifestations of lysosomal storage diseases (LSDs) has remained a major challenge because of its inability to deliver therapeutic agents efficiently across the intact blood-brain barrier. Non-specific therapies such as hematopoietic stem cell transplantation have been useful in globoid cell leukodystrophy (Krabbe disease) and in some mucopolysaccharidoses," scientists in the United States report.
"Anti-inflammatory agents also show promise as adjuvant therapy. High doses...
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Source: Gene Therapy Weekly (2010-08-05)
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