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Sanofi Genzyme focuses on developing specialty treatments for debilitating diseases that are often difficult to diagnose and treat, providing hope to patients and ...
ERTs work by breaking down the fatty substance that accumulates in peripheral ... acid sphingomyelinase deficiency (ASMD), which currently has no treatments ...
There's no force as strong as a mom on a mission. After learning of her son Ryker's diagnosis of #PompeDisease, Melanie McKay got to work. She founded ...
Sanofi Genzyme, Cambridge, Massachusetts. ... world, where the vast majority of people with hemophilia have limited or no access to diagnosis and treatment.