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iECURE's Treatment Achieves Breakthrough in Infant OTC Deficiency

1/11/2025

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Summary

Story summary

In a groundbreaking development, iECURE has achieved a complete clinical response in the first infant treated with ECUR-506 for neonatal onset ornithine transcarbamylase (OTC) deficiency. This promising treatment, which has been well tolerated, allowed the infant to stop ammonia scavenger medication and normalize protein intake. Full results are anticipated in early 2026 as enrollment continues across multiple countries.

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Story summary

In a groundbreaking development, iECURE has achieved a complete clinical response in the first infant treated with ECUR-506 for neonatal onset ornithine transcarbamylase (OTC) deficiency. This promising treatment, which has been well tolerated, allowed the infant to stop ammonia scavenger medication and normalize protein intake. Full results are anticipated in early 2026 as enrollment continues across multiple countries.