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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Main Authors: Stefano Vavassori (Author), Sean Russell (Author), Celeste Scotti (Author), Stefano Benvenuti (Author)
Format: Book
Published: Frontiers Media S.A., 2024-08-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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