Enzyme Replacement Therapy

New enzyme replacement therapy findings from University College described

Published in Gene Therapy Weekly, January 21st, 2010

"We analysed 5-year treatment with agalsidase alfa enzyme replacement therapy in patients with Fabry's disease who were enrolled in the Fabry Outcome Survey observational database (FOS). Baseline and 5-year data were available for up to 181 adults (126 men) in FOS," scientists in London, the United Kingdom report.

"Serial data for cardiac mass and function, renal function, pain, and quality of life were assessed. Safety and sensitivity analyses were done in patients with baseline and at least one relevant follow-up measurement during the 5 years (n=555 and n=475, respectively). In patients with baseline cardiac hypertrophy, treatment resulted in a sustained reduction in...

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Source: Gene Therapy Weekly (2010-01-21)

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