Published in Blood Weekly, September 11th, 2003
"In the last three decades, hemophilia has moved from the status of a neglected and often fatal hereditary disorder to that of a fully defined group of molecular-pathological entities for which safe and effective treatment is available. Hemophilia is likely to be the first widespread severe genetic condition to be cured by gene therapy in the third millennium," according to recent research from Italy.
"In the socio-economic arena it remains a challenge to humanity to know that four-fifths of the world's hemophiliacs still receive no treatment at all," noted P.M. Mannucci,...
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