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Sanofi’s investigational drug, with a track record of mixed results in the clinic, has passed a Phase 3 trial in Gaucher disease, but flunked a separate late-stage study in Fabry disease.
The French drugmaker said on Monday that venglustat met the primary endpoint in the 43-patient LEAP2MONO study, achieving “statistically significant improvements” in neurological symptoms of type 3 Gaucher disease at 52 weeks compared with standard-of-care enzyme replacement therapy (ERT).
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