Unlocking the full potential of rare disease drug development: exploring the not-for-profit sector's contributions to drug development and access

• Hoofdauteurs: Stefano Vavassori (Auteur), Sean Russell (Auteur), Celeste Scotti (Auteur), Stefano Benvenuti (Auteur)
• Formaat: Boek
• Gepubliceerd in: Frontiers Media S.A., 2024-08-01T00:00:00Z.
• Onderwerpen: article
• Online toegang: Connect to this object online.

This commentary provides a comprehensive overview of the challenges and opportunities in the field of drug development for rare diseases and especially of gene therapy products for ultra-rare diseases. It discusses the limited market size, reimbursement and scientific complexities that deter pharmac...