Genzyme, A Sanofi Company

This large international study tested whether the drug ZD6474 (Zactima) could slow tumor growth in people with advanced medullary thyroid cancer that couldn't be removed by surgery. Researchers compared the drug against a placebo in 331 patients to see if it could delay cancer pr…

This study tested the safety and effectiveness of Aldurazyme, an enzyme replacement therapy, in 12 Chinese patients with Mucopolysaccharidosis I (MPS I), a rare genetic disorder. Participants received weekly infusions for 26 weeks. The main goals were to check for side effects an…

This study tested the safety and effectiveness of Fabrazyme, an enzyme replacement therapy, for Chinese patients with Fabry disease. Twenty-two participants received treatment for 48 weeks while researchers monitored side effects and changes in disease markers. The goal was to un…

This study created a large international registry to collect health information from over 1,400 patients with Mucopolysaccharidosis I (MPS I), a rare genetic disorder. The goal was to better understand how the disease naturally progresses and how patients respond to existing trea…

This study aimed to understand how the body absorbs, breaks down, and eliminates the experimental drug venglustat. Six healthy male volunteers received a single, traceable dose. Researchers then measured how much of the drug and its byproducts were excreted in urine and feces ove…