Genzyme, A Sanofi Company

This study tested an experimental drug called venglustat in adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that damage the brain and nerves. The goal was to see if daily pills could slow the disease by reducing harmful fatty buildup. The…

This study tested an experimental drug called lademirsen in 43 adults with Alport syndrome, a genetic condition that damages kidneys. The goal was to see if weekly injections could slow kidney function decline and check for side effects. The trial was terminated early, but resear…

This study aimed to see if the drug alemtuzumab could help children aged 10 to 17 with relapsing-remitting multiple sclerosis (MS) whose disease was still active despite other treatments. Researchers planned to measure brain lesions and safety. The trial was terminated early, so …