Invest Like the Best with Patrick O'Shaughnessy cover image

Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428]

Invest Like the Best with Patrick O'Shaughnessy

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Navigating Gene Therapy Challenges in Rare Diseases

This chapter explores the intricacies of gene therapy in relation to spinal muscular atrophy, emphasizing the need for backup genes and optimal therapeutic windows. It also reflects on the historical funding landscape for rare diseases and the pressing concerns of parents advocating for research and treatment options.

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