Onasemnogene abeparvovec, gene therapy, and the hierarchy of evidence
Play • 9 min
Gene therapy - it's here, and it's really expensive! What does onasemnogene abeparvovec do? How does how it works compare to other (also complicated and expensive) medicines? How strong can the evidence be if it's not from a randomised controlled trial? And, probably most importantly, why on earth does it have 10 syllables in its name and how might you go about butchering its pronunciation?
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