Communiqué de presse : Le venglustat de Sanofi a atteint tous les critères d'évaluation principaux dans une étude de phase 3 sur la maladie de Gaucher de type 3

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• These results underscore Sanofi's commitment to rare disease research and desire to offer new perspectives to people living with these conditions

  80% confidence

• A daily pill could make a considerable difference for Gaucher disease patients facing neurological challenges

  80% confidence

• PERIDOT study showed pain reduction in both study arms and did not meet the primary endpoint

  80% confidence

• Venglustat was generally well tolerated with no new safety signals compared to previous studies

  80% confidence

• Venglustat achieved equivalent performance to enzyme replacement therapy on non-neurological criteria including spleen volume, liver volume, and hemoglobin levels

  80% confidence

• There is currently no approved treatment for the neurological manifestations of GD3

  80% confidence

• Venglustat demonstrated statistically significant improvements in neurological symptoms (p=0.007) compared to enzyme replacement therapy at week 52

  80% confidence

Data points we hold from this source