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

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...

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Asıl Yazarlar: Stefano Vavassori (Yazar), Sean Russell (Yazar), Celeste Scotti (Yazar), Stefano Benvenuti (Yazar)
Materyal Türü: Kitap
Baskı/Yayın Bilgisi: Frontiers Media S.A., 2024-08-01T00:00:00Z.
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3rd Floor Main Library

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