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  • Zolgensma shows 94% effect during post-evaluations
  • by Lee, Tak-Sun | translator Alice Kang | Jan 6, 2025 05:56am
¡¦ only 1 out of 18 patients fail treatment
17 showed significant improvement...mostly in infants and young children
The spinal muscular atrophy (SMA) treatment Zolgensma (onasemnogene abeparvovec) showed promising results suited to its high price.

The use of the once-daily, one-shot treatment, which has an insurance cap set at KRW 2, requires a post-marketing evaluation to measure its cost-effectiveness.

According to industry sources on the 5th, Zolgensma Inj was approved for reimbursement in August 2022 and was administered to 21 patients by November last year, 18 of whom have received outcome evaluations.

17 of the 18 patients showed significant improvement, with 1 patient failing to receive the drug due to death.

The Health Reviews and Assessment Service recently released the results of the Zolgensma Inj performa
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