Genzyme, A Sanofi Company

This study tested a drug called Fabrazyme in 22 Chinese patients with Fabry disease, a rare genetic condition that causes pain and organ damage. Patients received enzyme replacement therapy for 54 weeks to see if it was safe and helped control the disease. The main focus was on s…

This study tested a drug called ZD6474 (vandetanib) in 331 people with advanced medullary thyroid cancer that could not be removed by surgery. The goal was to see if the drug could slow tumor growth compared to a placebo. Results focused on how long patients lived without their c…

This study looked at the safety and effectiveness of Aldurazyme (laronidase) in 12 Chinese people with MPS I, a rare genetic disorder. Participants received weekly infusions for 26 weeks. The main goals were to track side effects and measure changes in urinary GAGs (a marker of d…

This study is a registry that collects information from over 1,400 people with MPS I, a rare genetic disorder. The goal is to learn how the disease progresses and how well treatments like enzyme replacement therapy work over the long term. No new treatments are given; participant…

This study looked at how a single dose of the experimental drug venglustat is processed and removed from the body in 6 healthy men. Researchers measured the drug and its breakdown products in blood, urine, and stool to understand its metabolism and excretion. The goal was to gath…