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Having enjoyed a protracted period of market monopoly, Genzyme - which pioneered enzyme replacement therapy for Gauchers disease - now faces the first real competition to Cerezyme in the form of two novel ERTs.
Gauchers disease arises from specific genetic mutations that correspond to distinct phenotypes of vastly different severity.
Genetic Diseases and Dysmorphic Syndromes - Gauchers Disease Drug Pipeline Report" contains detailed information on the current drug pipeline.
Top 20 companies in late stage Gauchers Disease Projects
Rosenthal has directed and performed extensive research on blood coagulation, hemorrhagic and thromboembolic diseases, leukemia and Gauchers Disease.