Genzyme, A Sanofi Company
Clinical trials sponsored by Genzyme, A Sanofi Company, explained in plain language.
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New hope for babies with pompe disease: stronger enzyme therapy tested in kids who need better control
Disease control OngoingThis study tests a new enzyme replacement therapy called avalglucosidase alfa in 22 children with infantile-onset Pompe disease. These children were already on standard treatment but were still getting worse or not improving enough. The goal is to see if the new drug is safe and …
Phase: PHASE2 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated May 26, 2026 11:27 UTC
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Pompe disease patients get continued access to enzyme therapy in french study
Disease control OngoingThis study gives Pompe disease patients in France continued access to the enzyme replacement therapy avalglucosidase alfa after they finished earlier clinical trials. It monitors long-term safety and how well the treatment works over time. About 17 adults and children with late-o…
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated May 22, 2026 13:53 UTC
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New drug shows promise in reducing bleeding for hemophilia patients
Disease control OngoingThis study looks at the long-term safety and effectiveness of a drug called fitusiran in people with hemophilia A or B, including those with inhibitors. About 281 participants who completed a previous fitusiran trial will receive the drug and be monitored for bleeding episodes an…
Phase: PHASE3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated May 20, 2026 11:57 UTC