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原文連結
論文資訊
- 類型：已發表論文
- 日期：2025
摘要
Therapeutic development for rare 疾病s is difficult for pharmaceutical companies due to significant scientific challenges, extensive costs, and low financial returns. It is increasingly common for caregivers and patient advocacy groups to partner with biomedical professionals to finance and develop treatments for rare 疾病s. This case study illustrates the story of Terry Pirovolakis, a father who partnered with biomedical professionals to develop the novel gene therapy, Melpida, within 36 months of the diagnosis of his infant son. We identify the factors that led to the success of Melpida and analyze the business model of Elpida Therapeutics, a 社會 purpose corporation founded by Pirovolakis to reproduce the success of Melpida for other rare 疾病s. We conclude with four lessons from Melpida to inf
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