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 who are failing standard therapy
Disease control OngoingThis study tests a new enzyme therapy called avalglucosidase alfa in 22 children with infantile-onset Pompe disease who are not doing well on the current standard treatment. The goal is to see if the new drug is safe and can better control the disease. Participants receive increa…
Phase: PHASE2 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated May 17, 2026 03:13 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 male participants aged 12 and older who completed a previous fitusiran trial will be followed. The goal is to see if …
Phase: PHASE3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated May 17, 2026 03:10 UTC
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French study tracks Long-Term effects of rare muscle disease treatment
Disease control OngoingThis study continues to provide and monitor an enzyme replacement therapy called avalglucosidase alfa for 17 patients in France with Pompe disease, a rare genetic disorder that weakens muscles. The purpose is to collect long-term safety data and see how well the treatment continu…
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Apr 20, 2026 16:15 UTC