# The baby girl was diagnosed with Canavan disease at birth.

*biotech · news · 2026-04-30 · The Irish Times*

## Key points

- An Irish child received experimental gene therapy for Canavan disease at Dayton Children’s Hospital in Ohio.
- Initial results show positive signs like reduced brain swelling and increased alertness within weeks of treatment.
- Prof Paola Leone’s team reported positive outcomes from eight children, including the Irish baby, in Nature Medicine.
- The biotech firm Myrtelle plans to seek FDA approval in 2024, with possible treatment approval by 2027.
- Earlier intervention with gene therapy appears critical, as older children may not benefit if brain damage has progressed.

**Companies:** Myrtelle
**Countries:** Ireland, United States, Australia, Chile, Argentina, Slovakia

[Read the full story on The Irish Times](https://www.irishtimes.com/science/2026/04/30/irish-baby-among-worlds-first-to-receive-experimental-gene-therapy/)

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