Genzyme, A Sanofi Company

This Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However…

This study tested the drug alemtuzumab in children aged 10 to 18 with relapsing-remitting multiple sclerosis (MS) whose disease was still active despite other treatments. The goal was to see if the drug could reduce new brain lesions and improve quality of life. However, the tria…

This phase 2 trial tested a weekly injection of lademirsen in 43 adults with Alport syndrome, a genetic condition that damages kidneys. The goal was to see if the drug could slow the decline in kidney function. The study was terminated early, so the results are limited and it's u…