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

• मुख्य लेखकों: Stefano Vavassori (लेखक), Sean Russell (लेखक), Celeste Scotti (लेखक), Stefano Benvenuti (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Frontiers Media S.A., 2024-08-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...