Genzyme, A Sanofi Company

This study is checking the long-term safety and effectiveness of a drug called fitusiran for people with severe hemophilia A or B. It involves about 281 male participants, aged 12 and older, who have already been in a previous fitusiran trial. The main goal is to see how well the…

This study is testing a new enzyme replacement drug called avalglucosidase alfa for children with a severe, life-threatening form of Pompe disease. It is for kids under 18 who are already receiving the standard treatment but are still getting worse or not responding well enough. …

This study provides continued access to enzyme replacement therapy for French patients with Pompe disease who completed earlier research trials. It follows 17 patients for approximately four years to monitor long-term safety and track how well the treatment maintains their walkin…