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Breakthrough: The stories behind Life-Changing Therapies with Dr. William Pao

Nucleate Podcast

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The Aang Family's Impact on SMA Research

This chapter explores the journey of the Aang family and the development of the RISD-PLAM drug for spinal muscular atrophy, illustrating the importance of patient advocacy in rare disease research. It highlights the challenges in securing funding, the role of private foundations, and the critical need for continued investment in scientific research to drive breakthroughs.

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