Genzyme, A Sanofi Company

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 large international study tested whether the drug ZD6474 could slow down advanced medullary thyroid cancer compared to a placebo. Researchers enrolled 331 patients with cancer that couldn't be surgically removed and measured how long the drug kept the cancer from worsening. …

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 track people with Mucopolysaccharidosis I (MPS I), a rare genetic disorder. It followed 1,406 patients to better understand how the disease naturally progresses and how treatments like enzyme replacement therapy work over the l…

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…