328: Pfizer's activist troubles, gene therapy questions, RFK Jr.'s MAHA ambitions
The Readout Loud
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Challenges in Duchenne Gene Therapy
This chapter explores the difficulties Pfizer faces with its Duchenne gene therapy program, spotlighting a failed clinical trial that raises questions about the use of microdystrophin as an approval biomarker. It also examines the broader implications for gene therapy evaluations, regulatory standards, and the uncertainty felt by families and physicians regarding effective treatments.
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