Gaucher Disease
Research from E.H. Davies et al broadens understanding of Gaucher disease
Published in Gene Therapy Weekly, December 13th, 2007
"Enzyme replacement is effective in managing the visceral disease; however, the neurological manifestations remain a more challenging obstacle. There is an unfulfilled need to reliably monitor neurological disease and its response to treatment. A severity scoring tool was developed through neurological domain identification, item generation and tool formation. Domain identification was...
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Source: Gene Therapy Weekly (2007-12-13)
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