Communiqué de presse : Le venglustat de Sanofi a reçu la désignation de thérapie innovante aux États-Unis pour la maladie de Gaucher de type 3

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• There is no approved treatment for neurological symptoms of Gaucher disease type 3

  60% confidence

• This regulatory milestone recognizes the significant unmet medical need for people living with Gaucher disease type 3, particularly those experiencing progressive neurological deterioration

  60% confidence

• Venglustat works by reducing abnormal accumulation of glycosphingolipids and is designed to cross the blood-brain barrier to target the underlying pathology causing neurological effects of GD3

  60% confidence

• Sanofi will continue to collaborate with the FDA to advance this potential therapeutic option

  60% confidence

• There is currently no approved treatment for the neurological symptoms of Gaucher disease type 3

  60% confidence

• This regulatory milestone recognizes the significant unmet medical need for people living with Gaucher disease type 3, particularly those experiencing progressive neurological deterioration

  60% confidence

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

  60% confidence

• The positive results from the LEAP2MONO study are an encouraging advancement in the research and development process

  60% confidence

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

  60% confidence

• Venglustat has the potential to slow the progression of Gaucher disease type 3 by inhibiting abnormal accumulation of glycosphingolipids and its pathophysiological consequences

  60% confidence

• The positive results from the LEAP2MONO study are an encouraging advance in the research and development process, and we will continue to collaborate with the FDA to advance this potential treatment option

  60% confidence