Communiqué de presse : Le Nexviazyme de Sanofi a atteint tous les critères d’évaluation principaux et secondaires dans une étude de phase III sur la forme infantile de la maladie de Pompe

O que extraímos desta fonte

The claims Via News extracted from this document. We point to the source; we don't replace it.

• Infantile-onset Pompe disease is a devastating and rapidly progressive condition presenting in the first days or weeks of life, making early intervention essential to improve survival free of invasive ventilation beyond one year; Baby-COMET shows the potential of avalglucosidase alfa to support ventilation-free survival in infants while achieving encouraging cardiac and motor outcomes.

  60% confidence

• The positive Baby-COMET results offer the potential to expand access to Nexviazyme for more patients and families facing a disease with limited therapeutic options during the earliest months of life; results are consistent with prior studies and reflect years of scientific research translating deep biological understanding into clinical advances for the Pompe disease community.

  60% confidence