Genzyme, A Sanofi Company

This phase 3 trial tested a daily oral drug called vandetanib (ZD6474) against a placebo in 331 people with advanced medullary thyroid cancer that could not be removed by surgery. The main goal was to see if the drug could delay tumor growth. Researchers also measured how many pa…

This study tested a drug called Aldurazyme (laronidase) in 12 Chinese people with mucopolysaccharidosis I (MPS I), a rare genetic disorder. Participants received weekly infusions for 26 weeks to see if the treatment is safe and reduces waste products in urine. The goal is to mana…

This study tested the safety and effectiveness of Fabrazyme (agalsidase beta) in 22 Chinese patients with Fabry disease, a rare genetic disorder. Participants received enzyme replacement therapy through IV infusions over 54 weeks. The study monitored side effects and changes in d…

This completed study tracked 42 women with multiple sclerosis who became pregnant around the time they received LEMTRADA infusions. Researchers aimed to measure rates of miscarriage and birth defects to better understand the drug's safety during pregnancy. The study did not test …

This registry tracked over 1,400 people with MPS I, a rare genetic disorder, to better understand how the disease progresses and how treatments like enzyme replacement therapy work over time. It did not test a new drug but collected real-world data to help doctors improve patient…

This early-stage study gave a single dose of the experimental drug venglustat to 6 healthy men to see how it is processed and eliminated from the body. Researchers measured radioactivity in urine, feces, and blood to map the drug's breakdown and excretion. The goal was to underst…