Genzyme, A Sanofi Company

This study tested a drug called alemtuzumab in children and teenagers (ages 10-17) with relapsing multiple sclerosis (MS) whose disease was still active despite being on other standard MS medications. The goal was to see if switching to alemtuzumab could better control their MS b…

This study tested whether a weekly injection called lademirsen could safely slow the decline of kidney function in adults with Alport syndrome, a genetic condition that damages the kidneys. It involved 43 participants who received either the drug or a placebo for nearly a year. T…

This study tested whether a daily oral pill called venglustat could slow the progression of late-onset Tay-Sachs and Sandhoff disease, rare and severe inherited disorders that damage the brain and nerves. It involved about 75 adults and some younger participants over a two-year t…