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Gene therapy – SMA — RACP Paediatrics MCQ

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ModerateGeneticsGene therapy – SMARACP Paediatrics

A child with spinal muscular atrophy type 1 receives a single IV infusion of a gene therapy (onasemnogene abeparvovec/Zolgensma) that delivers a functional SMN1 gene via an AAV9 vector. What category of treatment is this?

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Correct answer: C — Gene therapy

Onasemnogene abeparvovec is a gene replacement therapy – it delivers a functional copy of the SMN1 gene via an adeno-associated virus (AAV9) vector as a single IV infusion. It is approved for SMA in children <2 years (some jurisdictions up to 21 kg). It has dramatically improved outcomes in SMA type 1, with many treated infants achieving motor milestones previously unachievable.

Reference: RACP Paediatric Curriculum – Genetics/Neurology; TGA – Zolgensma approval