Genzyme, A Sanofi Company

This study tested the drug ZD6474 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 cancer getting wors…

This study tested the safety and effectiveness of Fabrazyme, an enzyme replacement therapy, in 22 Chinese patients with Fabry disease. Fabry disease is a rare genetic disorder that causes pain, organ damage, and other serious symptoms. Patients received Fabrazyme infusions for 54…

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. Researchers measured side effects, changes in urine GAG levels, and liver size to see if the…

This study gave 6 healthy men a single dose of venglustat with a tiny radioactive marker to see how the drug is absorbed, broken down, and eliminated. Researchers measured radioactivity in blood, urine, and stool to understand the drug's path through the body. The goal was to lea…

This study is a registry that collects health information from people with MPS I, a rare genetic disorder. It does not test any new treatments but instead observes how the disease progresses and how current therapies work over the long term. Over 1,400 patients have participated,…